Publications by authors named "Anne Sirvent"

42 Publications

Medication non-adherence after allogeneic hematopoietic cell transplantation in adult and pediatric recipients: a cross sectional study conducted by the Francophone Society of Bone Marrow Transplantation and Cellular Therapy.

Fundam Clin Pharmacol 2020 Aug 2. Epub 2020 Aug 2.

Hematologie Adulte, CHU Lille, Lille, F 59000, France.

Medication non-adherence (NA) after allogeneic hematopoietic cell transplantation (allo-HCT) can lead to serious complications. This study assesses NA in French adult and pediatric recipients and identifies factors associated with NA. In accordance with the EMERGE and STROBE guidelines, a cross sectional multicentric survey was conducted. We used a self-reported questionnaire that was adapted to adults and pediatrics and that could provide a picture of all three phases of medication adherence: initiation, implementation, persistence. We enrolled 242 patients, 203 adults (mean age: 51 years old, 50.7% male) and 39 children (mean age: 9 years old, 56.4% female). Reported NA was estimated at about 75% in both populations, adults and pediatrics. In adults, the univariate analysis showed that patients less than 50 years old (P = 0.041), (i) treated with cyclosporine (P = 0.02), (ii) treated with valacyclovir/acyclovir (P = 0.016), and (iii) experiencing side effects (P = 0.009), were significantly more non-adherent. In multivariate analysis, only recipient age was significantly associated to NA (P = 0.05). The limited size of the pediatric population did not allow us to draw any statistical conclusion about this population. To the best of our knowledge, this is the first study in France on NA in allo-HCT recipients. Our results highlight the age factor as the only factor related to NA. Further studies are needed to confirm our observations and refine results in pediatric populations, currently most at risk of medication NA.
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http://dx.doi.org/10.1111/fcp.12593DOI Listing
August 2020

A Retrospective Comparison of DLI and gDLI for Post-Transplant Treatment.

J Clin Med 2020 Jul 12;9(7). Epub 2020 Jul 12.

Department of Clinical Haematology, CHU Montpellier, 34295 Montpellier, France.

Donor lymphocyte infusion (DLI) is used to prevent or treat haematological malignancies relapse after allogeneic stem cell transplantation (allo-SCT). Recombinant human granulocyte colony-stimulated factor primed DLI (gDLI) is derived from frozen aliquots of the peripheral blood stem cell collection. We compared the efficacy and safety of gDLI and classical DLI after allo-SCT. We excluded haploidentical allo-SCT. Initial diseases were acute myeloblastic leukaemia ( = 45), myeloma ( = 38), acute lymphoblastic leukaemia ( = 20), non-Hodgkin lymphoma ( = 10), myelodysplasia ( = 8), Hodgkin lymphoma ( = 8), chronic lymphocytic leukaemia ( = 7), chronic myeloid leukaemia ( = 2) and osteomyelofibrosis ( = 1). Indications for DLI were relapse ( = 96) or pre-emptive treatment ( = 43). Sixty-eight patients had classical DLI and 71 had gDLI. The response rate was 38.2%, the 5-year progression-free survival (PFS) rate was 38% (29-48) and the 5-year overall survival (OS) rate was 37% (29-47). Graft versus host disease rate was 46.7% and 10.1% of patients died from toxicity. There were no differences between classical DLI and gDLI in terms of response ( = 0.28), 5-year PFS ( = 0.90), 5-year OS (. 0.50), GvHD ( = 0.86), treated GvHD ( = 0.81) and cause of mortality (. 0.14). In conclusion, this study points out no major effectiveness or toxicity of gDLI compared to classical DLI.
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http://dx.doi.org/10.3390/jcm9072204DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7408819PMC
July 2020

Efficacy of a Hybrid Toothbrush versus Comparative Manual Toothbrush for Plaque Removal - Randomized In-Use Study.

Clin Cosmet Investig Dent 2020 29;12:241-250. Epub 2020 Jun 29.

Innovation Unit, Medical Department, Pierre Fabre Consumer Health Care, Castres, France.

Purpose: An innovative hybrid toothbrush was designed to function either in manual sonic mode or combined mode (manual and sonic). The primary objective was to assess the efficacy of a new hybrid powered toothbrush (PTB) used in combined mode versus a comparative manual toothbrush (MTB) for plaque removal, after 14 days of twice-daily use under normal conditions. The secondary objectives were to evaluate the gingival state, to evaluate the tolerance of the hybrid PTB and to evaluate its acceptability.

Materials And Methods: This study was a monocentric, block-randomized, dual treatment, parallel group, and examiner-blinded trial with before and after evaluation. It was conducted on two groups of 55 subjects presenting a visible plaque accumulation (score ≥2 as measured by the Turesky Modification of the Quigley-Hein Plaque Index (TMQHPI)). On Day 1/Day 8/Day 15, the same investigator conducted blind clinical examinations on each subject and evaluated TMQHPI and Papillary Bleeding Score (PBS). On Day 1, the subjects received either the hybrid PTB or the comparative MTB and used it twice daily under normal conditions of use.

Results: The hybrid PTB used in its combined mode eliminates dental plaque more efficiently than the comparative MTB, especially in difficult-to-access areas such as posterior and interproximal dental surfaces, while remaining gentle on the gingivae. The PBS was significantly lower with the hybrid toothbrush compared with the reference manual one.

Conclusion: The new device confirmed previous findings and should improve oral hygiene following the manufacturer's instructions. Moreover, the specific design of the toothbrush means that it can be used according to the oral environment conditions and personal feeling.
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http://dx.doi.org/10.2147/CCIDE.S257411DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7334036PMC
June 2020

Ruxolitinib in children with steroid-refractory acute graft-versus-host disease: A retrospective multicenter study of the pediatric group of SFGM-TC.

Pediatr Blood Cancer 2020 09 2;67(9):e28233. Epub 2020 Jul 2.

Department of Pediatric Hematology and Oncology, University Hospital of Rennes, Rennes, France.

Background: We conducted a national multicenter retrospective study in France to evaluate the efficacy and tolerance of ruxolitinib in children with steroid-refractory acute graft-versus-host disease (aGVHD) after allogeneic hematopoietic stem cell transplant.

Procedure: Patients were recruited from the 15 pediatric transplantation centers. Transplanted patients were eligible if they met the following criteria: aged ≤ 18 years at transplantation, receiving a myeloablative allogeneic hematopoietic stem cell transplant, having an aGVHD of grade ≥2, and treated with ruxolitinib for steroid-refractory aGVHD.

Results: Twenty-nine patients received ruxolitinib for steroid-refractory aGVHD. Six patients achieved a complete response at day 28 after the start of treatment but finally 19 patients (65.5%) achieved a complete response (CR) with a median delay of 41 days (5-93 days). Two patients had a partial response. All patients who achieved CR or partial response discontinued corticosteroid treatment. Eight patients showed treatment failure. The overall response rate was 72.4%. Twenty-three of 29 patients were alive at a median follow-up of 685 days (177-1042 days) after the hematopoietic stem cell transplantation. Viral replication was observed in 41.4% of cases. We did not observe severe hematological adverse events and cytopenia requiring a modification of ruxolitinib doses always resolved. The median initial dose of ruxolitinib was 12.6 mg/m /day with an important range. We could not demonstrate any relationship between initial dose and effectiveness.

Conclusion: Ruxolitinib may constitute a promising second-line treatment for children with steroid-refractory aGVHD that should be validated in a prospective large-scale pharmacokinetic and efficacy trial.
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http://dx.doi.org/10.1002/pbc.28233DOI Listing
September 2020

Haploidentical transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT Severe Aplastic Anemia Working Party.

Bone Marrow Transplant 2020 06 16;55(6):1050-1058. Epub 2019 Dec 16.

Hematology-Transplantation Department, Saint-Louis Hospital, Paris, France.

In the absence of an HLA-matched donor, the best treatment for acquired aplastic anemia patients refractory to immunosuppression is unclear. We collected and analyzed data from all acquired aplastic anemia patients who underwent a haploidentical transplantation with posttransplant cyclophosphamide in Europe from 2011 to 2017 (n = 33). The cumulative incidence of neutrophil engraftment was 67% (CI: 51-83%) at D +28 and was unaffected by age group, stem cell source, ATG use, or Baltimore conditioning regimen. The cumulative incidence of grades II-III acute GvHD was 23% at D +100, and limited chronic GvHD was 10% (0-20) at 2 years, without cases of grade IV acute or extensive chronic GvHD. Two-year overall survival was 78% (64-93), and 2-year graft-versus-host disease-free survival was 63% (46-81). In univariate analysis, the 2-year OS was higher among patients who received the Baltimore conditioning regimen (93% (81-100) versus 64% (41-87), p = 0.03), whereas age group, stem cell source, and ATG use had no effect. Our results using unmanipulated haploidentical transplantation and posttransplant cyclophosphamide for treating refractory AA patients are encouraging, but warrant confirmation in a prospective study with a larger number of patients and longer follow-up.
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http://dx.doi.org/10.1038/s41409-019-0773-0DOI Listing
June 2020

A high-emollient liquid cleanser for very dry and atopic-prone skin: Results of an in-use tolerance and efficacy study conducted under dermatological, pediatric, and ophthalmological supervision.

J Cosmet Dermatol 2020 May 27;19(5):1155-1160. Epub 2019 Nov 27.

Pierre Fabre Dermatologie, Lavaur, France.

Background: Emollients play a key role in the treatment of eczematous lesions and xerosis such as in atopic dermatitis. However, studies that show the actual benefits of cleansers are few and far between.

Aims: This study aims to evaluate the tolerance and efficacy of a high-emollient liquid cleanser (HELC) designed for very dry and atopic-prone skin, in the absence of any additional skin care. The product is a soap-free and fragrance-free liquid cleanser, containing mild surfactants and a ternary system of selected emollients: glycerin, vaseline, and paraffin.

Methods: In-use study was conducted under dermatological, pediatric, and ophthalmological supervision in 50 subjects (infants, children, and adults) with "dry to very dry and atopic-prone" skin. The primary objective of this monocentric, open, and intra-individual study was to assess the dermatological and ophthalmological tolerance of HELC after 21 days of using it at least once a day on the face and body. The secondary objectives were to evaluate its efficacy based on a clinical score (SCORAD), assess its short- and long-term moisturizing effect by measuring hydration rates (Corneometer ), and ascertain its cosmetic acceptability through a subjective evaluation questionnaire.

Results: The study validates the good dermatological and ophthalmological tolerance of HELC. Its efficacy was demonstrated by improvements in the SCORAD and moisturizing scores. Furthermore, the product was very well accepted by the subjects.

Conclusion: The fragrance-free HELC tested in this study for 21 days on "dry to very dry and atopic-prone skin" improves skin dryness and pruritus while ensuring good tolerance.
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http://dx.doi.org/10.1111/jocd.13141DOI Listing
May 2020

Outcomes of Salvage Haploidentical Transplant with Post-Transplant Cyclophosphamide for Rescuing Graft Failure Patients: a Report on Behalf of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy.

Biol Blood Marrow Transplant 2019 09 24;25(9):1798-1802. Epub 2019 May 24.

Bone Marrow Transplant Unit, Saint Louis Hospital, Paris, France.

Prognosis of patients with graft failure is dismal, and retransplantation is the sole option for long-term survival. To address the interest of haploidentical transplantation as a salvage option in this context, we analyzed data from 24 patients with graft failure or loss retransplanted with a haploidentical donor who received post-transplant cyclophosphamide (PTCy) as graft-versus-host disease prophylaxis (GVHD). Fludarabine-based reduced-intensity conditioning was used in 23 patients and the Baltimore regimen in 14 patients. The median delay between previous and salvage transplantation for graft failure was 63 days (range, 39 to 98). In addition to PTCy, all patients received cyclosporine, and 22 patients also received mycophenolate mofetil for GVHD prophylaxis. With a median follow-up of 353 days (range, 16 to 2010), 1-year overall survival (OS) was 56% (95% confidence interval, 38% to 81%). Transplant complications accounted for 80% of deaths. The cumulative incidence of neutrophil engraftment at day +30 was 79%. Cumulative incidence of grades II to IV acute GVHD at day 100 was 14%, and 1-year cumulative incidence of chronic GVHD was 31%. One-year cumulative incidence of relapse was 13%. Stem cell source did not impact on engraftment, GVHD, relapse, or OS. Salvage haploidentical transplant with PTCy for rescuing graft failure patients leads to an acceptable 1-year OS and might be a valid option in this poor situation.
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http://dx.doi.org/10.1016/j.bbmt.2019.05.013DOI Listing
September 2019

[Children and adolescents discharge guidelines after allogeneic stem cell transplantation for healthcare professionals: Recommendations of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC)].

Bull Cancer 2020 Jan 15;107(1S):S62-S67. Epub 2019 Apr 15.

Hôpital Robert-Debré, département d'hémato-immunologie pédiatrique, 48, boulevard Serurier, 75019 Paris, France; Université Paris 7-Paris Diderot, 5, rue Thomas-Mann, 75013 Paris, France. Electronic address:

Recommendations for visits or environment restrictions, and sometimes for food are usually well described for inpatient within HSCT unit procedures where those measures are less precise and detailed for outpatient from the discharge to the immune reconstitution achievement. The present paper main objective is to define risk patient groups depending on time, immune-suppressive drugs as well as graft-versus-host disease and immune reconstitution. We define here 3 risk patient groups and propose measures about house cleaning, pets, schools, social activities, hygiene, foods, sexual life and siblings.
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http://dx.doi.org/10.1016/j.bulcan.2019.03.006DOI Listing
January 2020

[Second allogeneic hematopoietic stem cell transplant: Guidelines from the francophone Society of bone marrow transplantation and cellular therapy (SFGM-TC)].

Bull Cancer 2019 Jan 6;106(1S):S40-S51. Epub 2018 Nov 6.

Cliniques universitaires Saint-Luc, service d'hématologie, 10, avenue Hippocrate, 1200 Bruxelles, Belgique.

Disease recurrence and graft dysfunction after allogeneic hematopoietic stem cell transplantation (allo-HSCT) currently remain among the major causes of treatment failure in malignant and non-malignant hematological diseases. A second allo-HSCT is a valuable therapeutic option to salvage those situations. During the 8th annual harmonization workshops of the french Society of bone marrow transplantation and cellular therapy (SFGM-TC), a designated working group reviewed the literature in order to elaborate unified guidelines on feasibility, indications, donor choice and conditioning in the case of a second allo-HSCT. In case of relapse, a second allo-HSCT with reduced intensity or non-myeloablative conditioning is a reasonable option, particularly in patients with a good performance status (Karnofsky/Lansky>80%), low co-morbidity score (EBMT score≤3), a longer remission duration after the first allo-HSCT (>6 months), and who present low disease burden at the time of second allo-HSCT. Matched related donors tend to be associated with better outcomes. In the presence of graft dysfunction (primary and secondary graft rejection), an immunoablative conditioning regimen is recommended. A donor change remains a valid option, especially in the absence of graft-versus-host disease after the first allo-HSCT.
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http://dx.doi.org/10.1016/j.bulcan.2018.05.018DOI Listing
January 2019

Single-Unit versus Double-Unit Umbilical Cord Blood Transplantation in Children and Young Adults with Residual Leukemic Disease.

Biol Blood Marrow Transplant 2019 04 29;25(4):734-742. Epub 2018 Oct 29.

Department of Pediatric Hematology-Oncology, APHM, Timone Hospital, Aix-Marseille University, Marseille, France; Department of Public Health -EA 3279 Research Unit, Marseille University Hospital, Aix-Marseille University, Marseille, France. Electronic address:

We previously reported in a French prospective randomized study that transplantation of 2 unrelated cord blood (UCB) units instead of 1 unit does not decrease the risk of transplantation failure but may enhance alloreactivity. Here we evaluated the influence of pretransplantation minimal residual disease (MRD) on leukemia relapse and survival after single- versus double-UCB transplantation (UCBT). Among 137 children and young adults who underwent UCBT in this randomized study, 115 had available data on MRD assessment done immediately before initiation of the pretransplantation conditioning regimen. MRD was considered positive at a level of ≥10, which was the case of 43 out of 115 patients. Overall, the mean 3-year survival probability was 69.1 ± 4.4%, and it was not significantly influenced by the MRD level: 70.7 ± 5.4% in MRD-negative (<10) patients (n = 72), 71.1 ± 9.4% in MRD-positive patients with 10 ≤ MRD <10 (n = 26) and 58.8 ± 11.9% in MRD-positive patients with ≥10 (n = 17). In the MRD-positive group, the mean risk of relapse was significantly lower in the double-UCBT arm compared with the single-UCBT arm (10.5 ± 7.2% versus 41.7 ± 10.4%; P = .025) leading to a higher mean 3-year survival rate (82.6 ± 9.3% versus 53.6 ± 10.3%; P = .031). This difference was observed only in patients who had not received antithymocyte globulin during their conditioning regimen. In the MRD-negative group, there was no differencebetween the single- and the double-UCBT arms. We conclude that even in cases of positive pretransplantation MRD, UCBT in children and young adults with acute leukemia yields a high cure rate, and that a double-unit strategy may enhance the graft-versus-leukemia effect and survival in these patients.
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http://dx.doi.org/10.1016/j.bbmt.2018.10.016DOI Listing
April 2019

A novel dermo-cosmetic product containing thermal spring water, sucralfate, copper sulfate, and zinc sulfate in the management of hand eczema.

Clin Cosmet Investig Dermatol 2018 16;11:373-381. Epub 2018 Jul 16.

Clinical Development Department, Pierre Fabre Dermo-Cosmetics, Toulouse, France,

Background: The regular use of cosmetic products plays a role in the management of hand eczema (HE) and aids in improving barrier function reducing dryness, roughness, pruritus and improving quality of life (QoL). The aim of this open-label study was to assess the efficacy and the reparative effect of a dermo-cosmetic product on subjects suffering from HE after 7 and 21 days of daily application.

Methods: The product was a water-in-oil (W/O) emulsion containing the active ingredients Avène thermal spring water, sucralfate, and copper and zinc sulfates. In total, 32 subjects suffering from either contact dermatitis or climatic dermatitis participated in the trial. The modified total lesion symptom score and physician global assessment scores were used to describe the severity of HE. The safety of the product was assessed through clinical scoring. The subjective tolerance, and acceptance, were documented using a self-assessment questionnaire completed by the subjects. The impact of the dermatosis on QoL was evaluated using the Dermatology Life Quality Index.

Results: After 7 days of application, both the physician and subjects noticed a significant improvement in HE. The formula was very well tolerated and accepted. These benefits were correlated with a significant improvement in QoL.

Conclusion: The W/O emulsion used in this study demonstrated real benefits for the subjects suffering from contact dermatitis and climatic dermatitis.
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http://dx.doi.org/10.2147/CCID.S157849DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6053173PMC
July 2018

Unrelated cord blood transplantation in patients with idiopathic refractory severe aplastic anemia: a nationwide phase 2 study.

Blood 2018 08 14;132(7):750-754. Epub 2018 May 14.

Bone Marrow Transplantation (BMT) Unit, Saint Louis Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.

Outcomes remain poor for refractory severe aplastic anemia (SAA) patients. Alternative donor transplantation may be considered, but results from previous studies are not encouraging. We conducted a prospective nationwide phase 2 study to assess unrelated cord blood (CB) transplantation (CBT) efficacy and safety in refractory SAA patients (Aplastic Anemia and Cord Blood Transplantation protocol). To demonstrate a significant difference in 1-year survival from 20% (null hypothesis) to 50% (alternative hypothesis), we needed to include 25 transplanted patients and therefore included 26 (median age, 16 years). Eligibility criteria required 1 or 2 unrelated CB units, containing separately or together >4 × 10 frozen nucleated cells (NCs) per kilogram of recipient body weight. Conditioning regimen comprised fludarabine (FLU), cyclophosphamide (CY), antithymocyte globulin (ATG), and 2-Gy total body irradiation (TBI). With a median follow-up of 38.8 months, engraftment occurred in 23 patients (88%); cumulative incidences of grade II-IV acute and chronic graft-versus-host disease were 45.8% and 36%, respectively. Twenty-three patients were alive at 1 year, with an 88.5% overall survival (OS) rate, differing significantly from the expected 20% ( < .0001; 84% OS at 2 years). CBT with units containing ≥4 × 10 frozen NCs per kilogram is therefore a valuable curative option for young adults with refractory SAA and no available matched unrelated donors. This trial was registered at www.clinicaltrials.gov as #NCT01343953.
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http://dx.doi.org/10.1182/blood-2018-01-829630DOI Listing
August 2018

Acute Myeloid Leukemia With Central Nervous System Involvement in Children: Experience From the French Protocol Analysis ELAM02.

J Pediatr Hematol Oncol 2018 01;40(1):43-47

Department of Pediatric Hematology and Oncology, Trousseau Hospital.

Central nervous system (CNS) involvement at diagnosis of pediatric acute myeloid leukemia (AML) is not considered as an independent prognostic factor. This study describes the prognostic value of pediatric AML with CNS involvement at diagnosis. Pediatric patients were treated for de novo AML in the French multicenter trial ELAM02. Lumbar puncture was carried out in the first week, and the treatment was adapted to the CNS status. No patient received CNS radiotherapy. The patients were classified into 2 groups: CNS+ and CNS-. Of the 438 patients, 16% (n=70) had CNS involvement at diagnosis, and 29% showed clinical signs. The patients with CNS disease were younger (40% were below 2 y old), had a higher white blood cell count (median of 45 vs. 13 G/L), and had M4 and M5 morphologies. The complete remission rate was similar at 92.8% for CNS+ and 88.5% for CNS-. There was no significant difference between the CNS+ and the CNS- group in overall survival (76% and 71%, respectively) and event-free survival (57% and 52%, respectively). Regarding the occurrence of first relapse, the CNS+ group had a higher combined relapse rate of 26.1% compared with 10% for the CNS- group. The results indicate that CNS involvement at diagnosis of pediatric AML is not an independent prognostic factor. Triple intrathecal chemotherapy combined with high-dose intravenous cytarabine should be the first-line treatment for CNS disease.
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http://dx.doi.org/10.1097/MPH.0000000000001034DOI Listing
January 2018

[Preservation/congelation of hematopoietic stem cell grafts in a pediatric context: Guidelines from the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC)].

Bull Cancer 2017 Dec 21;104(12S):S136-S141. Epub 2017 Nov 21.

Université Paris 7, hôpital Robert-Debré, service d'hémato-immunologie, 75019 Paris, France. Electronic address:

The Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC) organized the 7th allogeneic hematopoietic stem cell transplantation clinical practices harmonization workshop series in September 2016 in Lille, France. The objective of our workshop is to provide a discussion on the conservation and congelation of hematopoietic stem cells in a pediatric setting as well as our recommendations for this technique.
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http://dx.doi.org/10.1016/j.bulcan.2017.04.008DOI Listing
December 2017

Flow cytometry minimal residual disease after allogeneic transplant for chronic lymphocytic leukemia.

Eur J Haematol 2017 Apr 30;98(4):363-370. Epub 2017 Jan 30.

Hématologie, Groupe Hospitalier Pitié-Salpêtrière, Paris, France.

Objectives: This study investigates whether achieving complete remission (CR) with undetectable minimal residual disease (MRD) after allogeneic stem cell transplantation (allo-SCT) for chronic lymphocytic leukemia (CLL) affects outcome.

Methods: We retrospectively studied 46 patients transplanted for CLL and evaluated for post-transplant MRD by flow cytometry.

Results: At transplant time, 43% of the patients were in CR, including one with undetectable MRD, 46% were in partial response, and 11% had refractory disease. After transplant, 61% of the patients achieved CR with undetectable MRD status. By multivariate analysis, reaching CR with undetectable MRD 12 months after transplant was the only factor associated with better progression-free survival (P = 0.02) and attaining undetectable MRD, independently of the time of negativity, was the only factor that correlated with better overall survival (P = 0.04).

Conclusion: Thus, achieving undetectable MRD status after allo-SCT for CLL is a major goal to improve post-transplant outcome.
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http://dx.doi.org/10.1111/ejh.12836DOI Listing
April 2017

[Assessment and management of post-transplant iron overload: Guidelines of the Francophone Society of Marrow Transplantation and Cellular Therapy (SFGM-TC)].

Bull Cancer 2016 Nov 11;103(11S):S255-S266. Epub 2016 Nov 11.

CHU de Liège, service d'hématologie clinique, 4000 Liège, Belgique. Electronic address:

To harmonize clinical practice in hematopoietic stem cell transplantation, the Francophone Society of Bone Marrow Transplantation and Cell Therapy (SFGM-TC) set up the sixth annual series of workshops which brought together practitioners from all member centers and took place in September 2015 in Lille. The main aim of this session was to describe the impact, evaluation and treatment of post-transplant iron overload.
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http://dx.doi.org/10.1016/j.bulcan.2016.09.003DOI Listing
November 2016

The Impact of Donor Type on Long-Term Health Status and Quality of Life after Allogeneic Hematopoietic Stem Cell Transplantation for Childhood Acute Leukemia: A Leucémie de l'Enfant et de L'Adolescent Study.

Biol Blood Marrow Transplant 2016 11 10;22(11):2003-2010. Epub 2016 Aug 10.

Department of Pediatric Hematology and Oncology, Timone Enfants Hospital and Aix-Marseille University, Marseille, France; Research Unit EA 3279 and Department of Public Health, Aix-Marseille University and Timone Hospital Marseille, France.

We compared the long-term impact of donor type (sibling donor [SD] versus matched unrelated donor [MUD] or umbilical cord blood [UCB]) on late side effects and quality of life (QoL) in childhood acute leukemia survivors treated with hematopoietic stem cell transplantation. We included 314 patients who underwent transplantation from 1997 to 2012 and were enrolled in the multicenter French Leucémie de l'Enfant et de L'Adolescent ("Leukemia in Children and Adolescents") cohort. More than one-third of the patients were adults at last visit; mean follow-up duration was 6.2 years. At least 1 late effect was observed in 284 of 314 patients (90.4%). The average number of adverse late effects was 2.1 ± .1, 2.4 ± .2, and 2.4 ± .2 after SD, MUD, and UCB transplantation, respectively. In a multivariate analysis, considering the SD group as the reference, we did not detect an impact of donor type for most sequelae, with the exception of increased risk of major growth failure after MUD transplantation (odds ratio [OR], 2.42) and elevated risk of osteonecrosis after UCB transplantation (OR, 4.15). The adults and children's parents reported comparable QoL among the 3 groups. Adult patient QoL scores were lower than age- and sex-matched French reference scores for almost all dimensions. We conclude that although these patients are heavily burdened by long-term complications, donor type had a very limited impact on their long-term health status and QoL.
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http://dx.doi.org/10.1016/j.bbmt.2016.08.004DOI Listing
November 2016

Single- vs double-unit cord blood transplantation for children and young adults with acute leukemia or myelodysplastic syndrome.

Blood 2016 06 20;127(26):3450-7. Epub 2016 Apr 20.

Department of Pediatric Hematology, AP-HP, Robert Debré Hospital, Paris Diderot University, Paris, France.

Transplantation of 2 unrelated cord blood (UCB) units instead of 1 has been proposed to increase the cell dose. We report a prospective randomized study, designed to compare single- vs double-UCB transplantation in children and young adults with acute leukemia in remission or myelodysplasia. Eligible patients had at least two 4-6 HLA-identical UCBs with >3 × 10(7) nucleated cells/kg for the first and >1.5 × 10(7) for the second. The primary end point was the 2-year cumulative incidence of transplantation strategy failure, a composite end point including transplant-related mortality (TRM), engraftment failure, and autologous recovery. Randomized patients who did not proceed to transplantation due to refractory disease were considered transplantation failures. A total of 151 patients were randomized and included in the intent-to-treat analysis; 137 were transplanted. Double-UCB transplantation did not decrease transplantation strategy failure (23.4% ± 4.9% vs 14.9% ± 4.2%). Two-year posttransplant survival, disease-free survival, and TRM were 68.8% ± 6.0%, 67.6% ± 6.0%, and 5.9% ± 2.9% after single-unit transplantation compared with 74.8% ± 5.5%, 68.1% ± 6.0%, and 11.6% ± 3.9% after double-unit transplantation. The final relapse risk did not significantly differ, but relapses were delayed after double-unit transplantation. Overall incidences of graft-versus-host disease (GVHD) were similar, but chronic GVHD was more frequently extensive after double-UCB transplantation (31.9% ± 5.7% vs 14.7% ± 4.3%, P = .02). In an exploratory subgroup analysis, we found a significantly lower relapse risk after double-unit transplantation in patients receiving total body irradiation without antithymocyte globulin (ATG), whereas the relapse risk was similar in the group treated with busulfan, cyclophosphamide, and ATG. Single-UCB transplantation with adequate cell dose remains the standard of care and leads to low TRM. Double-unit transplantation should be reserved for patients who lack such units. This trial was registered at www.clinicaltrials.gov as #NCT01067300.
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http://dx.doi.org/10.1182/blood-2016-01-694349DOI Listing
June 2016

Late thyroid complications in survivors of childhood acute leukemia. An L.E.A. study.

Haematologica 2016 06 11;101(6):747-56. Epub 2016 Mar 11.

Department of Pediatric Hematology and Oncology, Timone Enfants Hospital and Aix-Marseille University, France Research Unit EA 3279 and Department of Public Health, Aix-Marseille University and Timone Hospital Marseille, France.

Thyroid complications are known side effects of irradiation. However, the risk of such complications in childhood acute leukemia survivors who received either central nervous system irradiation or hematopoietic stem cell transplantation is less described. We prospectively evaluated the incidence and risk factors for thyroid dysfunction and tumors in survivors of childhood acute myeloid or lymphoid leukemia. A total of 588 patients were evaluated for thyroid function, and 502 individuals were assessed for thyroid tumors (median follow-up duration: 12.6 and 12.5 years, respectively). The cumulative incidence of hypothyroidism was 17.3% (95% CI: 14.1-21.1) and 24.6% (95% CI: 20.4-29.6) at 10 and 20 years from leukemia diagnosis, respectively. Patients who received total body irradiation (with or without prior central nervous system irradiation) were at higher risk of hypothyroidism (adjusted HR: 2.87; P=0.04 and 2.79, P=0.01, respectively) as compared with transplanted patients who never received any irradiation. Patients transplanted without total body irradiation who received central nervous system irradiation were also at higher risk (adjusted HR: 3.39; P=0.02). Patients irradiated or transplanted at older than 10 years of age had a lower risk (adjusted HR: 0.61; P=0.02). Thyroid malignancy was found in 26 patients (5.2%). Among them, two patients had never received any type of irradiation: alkylating agents could also promote thyroid cancer. The cumulative incidence of thyroid malignancy was 9.6% (95% CI: 6.0-15.0) at 20 years. Women were at higher risk than men (adjusted HR: 4.74; P=0.002). In conclusion, thyroid complications are frequent among patients who undergo transplantation after total body irradiation and those who received prior central nervous system irradiation. Close monitoring is thus warranted for these patients. Clinicaltrials.gov identifier: NCT 01756599.
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http://dx.doi.org/10.3324/haematol.2015.140053DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5013950PMC
June 2016

Haematopoietic stem cell transplantation for refractory Langerhans cell histiocytosis: outcome by intensity of conditioning.

Br J Haematol 2015 Jun 27;169(5):711-8. Epub 2015 Mar 27.

CIBMTR® (Center for International Blood and Marrow Transplant Research), Department of Medicine, Medical College of Wisconsin, Milwaukee, WI, USA.

Patients with Langerhans cell histiocytosis (LCH) refractory to conventional chemotherapy have a poor outcome. There are currently two promising treatment strategies for high-risk patients: the first involves the combination of 2-chlorodeoxyadenosine and cytarabine; the other approach is allogeneic haematopoietic stem cell transplantation (HSCT). Here we evaluated 87 patients with high-risk LCH who were transplanted between 1990 and 2013. Prior to the year 2000, most patients underwent HSCT following myeloablative conditioning (MAC): only 5 of 20 patients (25%) survived with a high rate (55%) of transplant-related mortality (TRM). After the year 2000 an increasing number of patients underwent HSCT with reduced intensity conditioning (RIC): 49/67 (73%) patients survived, however, the improved survival was not overtly achieved by the introduction of RIC regimens with similar 3-year probability of survival after MAC (77%) and RIC transplantation (71%). There was no significant difference in TRM by conditioning regimen intensity but relapse rates were higher after RIC compared to MAC regimens (28% vs. 8%, P = 0·02), although most patients relapsing after RIC transplantation could be salvaged with further chemotherapy. HSCT may be a curative approach in 3 out of 4 patients with high risk LCH refractory to chemotherapy: the optimal choice of HSCT conditioning remains uncertain.
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http://dx.doi.org/10.1111/bjh.13347DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4433436PMC
June 2015

Use of Eculizumab in Patients With Allogeneic Stem Cell Transplant-Associated Thrombotic Microangiopathy: A Study From the SFGM-TC.

Transplantation 2015 Sep;99(9):1953-9

1 Hématologie Greffe de Moelle, Hôpital Saint Louis, APHP, Université Paris Diderot, Paris, France. 2 Service d'Hématologie Pédiatrique, Hôpital de la Timone, Marseille, France. 3 Service d'Onco-hematologie Pédiatrique, Hôpital Arnaud de Villeneuve, Centre Hospitalier Universitaire de Montpellier, Montpellier, France. 4 Service d'Hématologie, CHU de Purpan, Toulouse, France. 5 Département de Néphrologie et Transplantation d'organe, CHU de Rangueil, Toulouse, France. 6 Service d'Hématologie, Hôpital de la Pitié Salpetrière, APHP, Université Pierre et Marie Curie, Paris. 7 Service de Thérapie Cellulaire et d'Hématologie Clinique, Hôpital d'Estaing, CHU Clermont Ferrand, Université d'Auvergne, Clermont-Ferrand, France. 8 Service d'Immuno-hématologie et Rhumatologie pédiatrique, Hôpital Necker-Enfants Malades, Laboratoire INSERM U768, Institut Imagine, Université Sorbonne Paris-cité, Paris, France. 9 Centre de Néphrologie et Transplantation Rénale, Hôpital de la Conception, APHM, Université d'Aix Marseille, Marseille, France. 10 Service d'Hématologie, Hôpital Hautepierre, Hôpitaux Universitaire de Strasbourg, Strasbourg, France. 11 Département de Biostatistiques, Hôpital Saint Louis, APHP, Université Paris Diderot, Paris, France. 12 Service d'Immunologie Biologique, Hôpital Européen George Pompidou, APHP, CHU Paris Ouest, Paris, France. 13 Service d'Hématologie Biologique, Hôpital Antoine Beclère, APHP, Université Paris Sud, Clamart, France. 14 Service d'Hématologie, Centre de Référence des Microangiopathies Thrombotiques, Hôpitaux Universitaires de l'Est Parisien, APHP, Université Pierre et Marie Curie, Paris, France.

Background: Thrombotic microangiopathy (TMA) occurring after allogeneic hematopoietic stem cell transplantation (HSCT) has a devastating prognosis. Response rates to current therapies (mainly plasma exchange) are unsatisfactory. Thrombotic microangiopathy after allogeneic HSCT shares similarities with atypical hemolytic uremic syndrome (aHUS) in the underlying pathomechanisms. Eculizumab has been associated with impressive results in aHUS.

Materials And Methods: We retrospectively analyzed 12 patients who received Eculizumab in France between 2010 and 2013 for severe post-HSCT TMA.

Results: All 12 patients had severe TMA with neurological and/or renal involvement. Fifty-eight percent were refractory to first-line plasma exchange. At the time of TMA diagnosis, infections were present in 50% of the patients and acute graft-versus-host disease in 33%. Patients were treated with Eculizumab according to the aHUS therapeutic scheme. With a median follow-up of 14 months, hematological response and overall survival were 50% and 33%, respectively. Active acute graft-versus-host disease at TMA diagnosis was the only factor associated with worse overall survival (P = 0.009).

Discussion: Response rate and overall survival after Eculizumab in our cohort compare favorably with previously published data in TMA after allogeneic HSCT. Prospective trials are warranted to confirm these results. Early initiation of Eculizumab may have a favorable effect on long-term renal function and further contribute to the prolongation of survival.
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http://dx.doi.org/10.1097/TP.0000000000000601DOI Listing
September 2015

Decreased nonrelapse mortality after unrelated cord blood transplantation for acute myeloid leukemia using reduced-intensity conditioning: a prospective phase II multicenter trial.

Biol Blood Marrow Transplant 2015 Mar 18;21(3):445-53. Epub 2014 Nov 18.

Service d'Hématologie, Eurocord Office, Hôpital Saint-Louis, Paris, France; Department of Haematology, Churchill Hospital, Oxford, United Kingdom. Electronic address:

A prospective phase II multicenter trial was performed with the aim to obtain less than 25% nonrelapse mortality (NRM) after unrelated cord blood transplantation (UCBT) for adults with acute myeloid leukemia (AML) using a reduced-intensity conditioning regimen (RIC) consisting of total body irradiation (2 Gy), cyclophosphamide (50 mg/kg), and fludarabine (200 mg/m(2)). From 2007 to 2009, 79 UCBT recipients were enrolled. Patients who underwent transplantation in first complete remission (CR1) (n = 48) had a higher frequency of unfavorable cytogenetics and secondary AML and required more induction courses of chemotherapy to achieve CR1 compared with the others. The median infused total nucleated cells (TNC) was 3.4 × 10(7)/kg, 60% received double UCBT, 77% were HLA mismatched (4/6), and 40% had major ABO incompatibility. Cumulative incidence of neutrophil recovery at day 60 was 87% and the cumulative incidence of 100-day acute graft-versus-host disease (II to IV) was 50%. At 2 years, the cumulative incidence of NRM and relapse was 20% and 46%, respectively. In multivariate analysis, major ABO incompatibility (P = .001) and TNC (<3.4 × 10(7)/kg; P = .001) were associated with increased NRM, and use of 2 or more induction courses to obtain CR1 was associated with increased relapse incidence (P = .04). Leukemia-free survival (LFS) at 2 years was 35%, and the only factor associated with decreased LFS was secondary AML (P = .04). In conclusion, despite the decreased NRM observed, other RIC regimens with higher myelosuppression should be evaluated to decrease relapse in high-risk AML. (EUDRACT 2006-005901-67).
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http://dx.doi.org/10.1016/j.bbmt.2014.11.009DOI Listing
March 2015

Low non-relapse mortality and long-term preserved quality of life in older patients undergoing matched related donor allogeneic stem cell transplantation: a prospective multicenter phase II trial.

Haematologica 2015 Feb 25;100(2):269-74. Epub 2014 Nov 25.

Hematology Department, University Hospital Center, Nantes CRNCA, UMR 892 INSERM - 6299 CNRS, and Université de Nantes, Faculté de Médecine, Nantes, France Hematology and Cellular Therapy Unit, AP-HP, Université Paris 6, Hôpital Saint Antoine, Paris, France.

Allogeneic transplantation is a challenge in patients of advanced age because of a high risk of non-relapse mortality and potential long-lasting impairment of health-related quality of life. The development of reduced-intensity conditioning regimens has allowed the use of allogeneic transplantation in this population, but the optimal regimen remains undefined. We conducted a multicenter phase II trial evaluating the safety and efficacy of a reduced-intensity conditioning regimen combining fludarabine, intravenous busulfan, and rabbit antithymocyte globulins in patients older than 55 years of age transplanted from matched-related donor. In addition, health-related quality of life was prospectively measured. Seventy-five patients with a median age of 60 years (range 55-70) were analyzed. Grade III-IV acute and extensive chronic graft-versus-host diseases were found in 3% and 27% of patients, respectively. The day 100 and 1-year non-relapse mortality incidences were 1% and 9%, respectively. The cumulative incidences of relapse, progression-free survival and overall survival at two years were 36%, 51% and 67%, respectively, with a median follow up of 49 months. Global health-related quality of life, physical functioning, emotional functioning, and social functioning were not impaired compared to baseline for more than 75% of the patients (75%, 81.4%, 82.3%, and 75%, respectively). Thirty-four of the 46 (74%) progression-free patients at one year were living without persistent extensive chronic graft-versus-host disease. We conclude that the reduced-intensity conditioning regimen combining fludarabine, intravenous busulfan, and rabbit antithymocyte globulins is well tolerated in patients older than 55 years with low non-relapse mortality and long-term preserved quality of life.
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http://dx.doi.org/10.3324/haematol.2014.113571DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4803127PMC
February 2015

Allogeneic hematopoietic stem cell transplantation for T-prolymphocytic leukemia: a report from the French society for stem cell transplantation (SFGM-TC).

Eur J Haematol 2015 Mar 13;94(3):265-9. Epub 2014 Sep 13.

CHU Hôtel Dieu, Nantes, France.

T-prolymphocytic leukemia (T-PLL), a rare aggressive mature T-cell disorder, remains frequently resistant to conventional chemotherapy. Studies have suggested that allogeneic hematopoietic stem cell transplantation (HSCT) might possibly serve to consolidate the response to initial chemotherapy. The current report summarizes the outcome of 27 T-PLL cases identified in the registry in French Society for stem cell transplantation (SFGM-TC). Prior to HSCT, 14 patients were in complete remission (CR), 10 in partial response, three refractory, or in progression. Following HSCT, 21 patients achieved CR as best response. With a median follow-up for surviving patients of 33 (range, 6-103) months, 10 patients are still alive in continuous CR. Overall survival and progression-free survival estimates at 3 yr were 36% (95% CI: 17-54%) and 26% (95% CI: 14-45%), respectively. The relapse incidence after HSCT was 47% occurring at a median of 11.7 (range, 2-24) months. Overall cumulative incidence of transplant-related mortality was 31% at 3 yr. These results suggest that HSCT may allow long-term survival in patients with T-PLL following induction treatment; however, it is associated with a significant rate of toxicity.
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http://dx.doi.org/10.1111/ejh.12430DOI Listing
March 2015

Autologous stem cell transplantation in mantle cell lymphoma: a report from the SFGM-TC.

Ann Hematol 2014 Feb;93(2):233-42

Autologous stem cell transplantation (ASCT) is considered as an attractive treatment option for young mantle cell lymphoma (MCL) patients. This retrospective SFGM-TC study analyzed the outcome of 500 MCL patients treated with ASCT and investigated parameters that may modify the outcome of patients who proceeded to ASCT upfront (n=396). For all patients, median age at ASCT was 56 years (range, 26-71). Median follow-up was 34 months. Three-year progression free survival (PFS) and overall survival (OS)were 63.5% [95 % CI, 58.7–68.6 %] and 79.5 % [95 % CI, 75.3–83.4 %], respectively. Median time from ASCT to relapse was 22 months (range, 0–136 m). For patients transplanted upfront and in multivariate analysis, age (HR=2 [1.2–3.4], p=.01, and HR=2.3 [1.2–4.5], p=.01), disease status at time of ASCT (HR=1.7 [1.1–2.6], p=.01 and HR=1.8 [1.1–3.1], p=.03), and use of rituximab (HR=0.5 [0.3–0.8], p=.002 and HR=0.5 [0.3–0.9], p=.01) were statistically predictive for both PFS and OS. Also, first line treatment including anthracycline and high-dose cytarabine followed by ASCT conditioned with TAM improved PFS. To conclude, this study suggests that ASCT in MCL can provide a high response rate but may not be sufficient to cure MCL even when ASCT is performed upfront, highlighting the need for innovative approaches before ASCT, aiming to increase complete response rate, and after ASCT, to maintain response.
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http://dx.doi.org/10.1007/s00277-013-1860-8DOI Listing
February 2014

Evolving strategies with immunomodulating drugs and tandem autologous/allogeneic hematopoietic stem cell transplantation in first line high risk multiple myeloma patients.

Exp Hematol 2013 Dec 29;41(12):1008-15. Epub 2013 Aug 29.

Hematology, Centre Hospitalier Lyon Sud, Pierre-Bénite, France. Electronic address:

We prospectively evaluated in high-risk myeloma patients the efficacy and toxicity of tandem autologous hematopoietic stem cell transplantation (auto-HSCT) followed by reduced-intensity conditioning (RIC) and allogeneic (allo)-HSCT with bortezomib and donor lymphocyte infusions introduction after allo-HSCT (group 1). Results were compared with results from tandem auto-RIC-allo-HSCT without bortezomib (group 2). Groups 1 and 2 were compared to matched patients not receiving allo-HSCT from the Intergroupe Francophone du Myélome prospective studies. Allo-HSCT groups included 25 patients (12 in group 1, 13 in group 2). All patients engrafted. There were 8 acute GVHD (7 grade II [3 in group 1], 1 grade III in group 1)] and 11 chronic GVHD (3 limited [in group 1], 8 extensive [1 in group 1]). Matched population included 36 controls for group 1 and 39 for group 2. After a median follow-up of 55 months (range, 3-142 months), median overall survival was not reached in group 1 versus 65 months (51-not reached [NR]) in its matched group (p = 0.027); it was 96 months (49-NR) in group 2 versus 91 months (32-NR) in its matched group (p = 0.77). Median progression-free survival was 49 months (29-NR) in group 1 and was 25 months (range, 21-35 months) in its matched group (p = 0.0045); it was 31 months (22-NR) in group 2 and 28 months (range, 21-40 months) in its matched group (p = 0.0776). Tandem auto-RIC-allo-HSCT including new molecules and immunomodulation after transplantation could be used as a first-line treatment for high-risk myeloma patients.
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http://dx.doi.org/10.1016/j.exphem.2013.08.003DOI Listing
December 2013

Autologous stem cell transplantation in mantle cell lymphoma: a report from the SFGM-TC.

Ann Hematol 2014 Feb 16;93(2):233-42. Epub 2013 Aug 16.

Department of Hematology, University Hospital, Nantes, France.

Autologous stem cell transplantation (ASCT) is considered as an attractive treatment option for young mantle cell lymphoma (MCL) patients. This retrospective SFGM-TC study analyzed the outcome of 500 MCL patients treated with ASCT and investigated parameters that may modify the outcome of patients who proceeded to ASCT upfront (n = 396). For all patients, median age at ASCT was 56 years (range, 26-71). Median follow-up was 34 months. Three-year progression free survival (PFS) and overall survival (OS) were 63.5 % [95 % CI, 58.7-68.6 %] and 79.5 % [95 % CI, 75.3-83.4 %], respectively. Median time from ASCT to relapse was 22 months (range, 0-136 m). For patients transplanted upfront and in multivariate analysis, age (HR = 2 [1.2-3.4], p = .01, and HR = 2.3 [1.2-4.5], p = .01), disease status at time of ASCT (HR = 1.7 [1.1-2.6], p = .01 and HR = 1.8 [1.1-3.1], p = .03), and use of rituximab (HR = 0.5 [0.3-0.8], p = .002 and HR = 0.5 [0.3-0.9], p = .01) were statistically predictive for both PFS and OS. Also, first line treatment including anthracycline and high-dose cytarabine followed by ASCT conditioned with TAM improved PFS. To conclude, this study suggests that ASCT in MCL can provide a high response rate but may not be sufficient to cure MCL even when ASCT is performed upfront, highlighting the need for innovative approaches before ASCT, aiming to increase complete response rate, and after ASCT, to maintain response.
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http://dx.doi.org/10.1007/s00277-013-1860-8DOI Listing
February 2014

Double umbilical cord blood transplantation for hematological malignancies: a long-term analysis from the SFGM-TC registry.

Exp Hematol 2013 Nov 2;41(11):924-33. Epub 2013 Jul 2.

Centre Hospitalier Lyon Sud, Hématologie 1G, Hospices Civils de Lyon, Pierre Bénite, France.

Allogeneic hematopoietic stem cell (HSC) transplantation is a curative treatment for many hematologic malignancies for which umbilical cord blood (UCB) represents an alternative source of HSCs. To overcome the low cellularity of one UCB unit, double UCB transplantation (dUCBT) has been developed in adults. We have analyzed the outcome of 136 patients who underwent dUCBT reported to the SFGM-TC registry between 2005 and 2007. Forty-six patients received myeloablative regimens, and 90 patients received reduced-intensity conditioning regimens. There were 84 cases of leukemia, 17 cases of non-Hodgkin lymphoma, 11 cases of myeloma, and 24 other hematologic malignancies. At transplantation, 40 (29%) patients were in complete remission. At day 60 after transplantation, the cumulative incidence of neutrophil recovery was 91%. We observed one UCB unit domination in 88% of cases. The cumulative incidence of day 100 acute graft-versus-host disease, chronic graft-versus-host disease, transplant-related mortality, and relapse at 2 years were 36%, 23%, 27%, and 28% respectively. After a median follow-up of 49.5 months, the 3-year probabilities of overall and progression-free survival were 41% and 35%, respectively, with a significant overall survival advantage when male cord engrafted male recipients. We obtained a long-term plateau among patients in complete remission, which makes dUCBT a promising treatment strategy for these patients.
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http://dx.doi.org/10.1016/j.exphem.2013.05.297DOI Listing
November 2013

New autoimmune diseases after cord blood transplantation: a retrospective study of EUROCORD and the Autoimmune Disease Working Party of the European Group for Blood and Marrow Transplantation.

Blood 2013 Feb 17;121(6):1059-64. Epub 2012 Dec 17.

Eurocord, Hôpital Saint Louis Assistance Publique des Hôpitaux de Paris (AP-HP), University Paris VII, Paris, France.

To describe the incidence, risk factors, and treatment of autoimmune diseases (ADs) occurring after cord blood transplantation (CBT), we analyzed both CBT recipients reported to EUROCORD who had developed at least 1 new AD and those who had not. Fifty-two of 726 reported patients developed at least 1 AD within 212 days (range, 27-4267) after CBT. Cumulative incidence of ADs after CBT was 5.0% +/- 1% at 1 year and 6.6% +/- 1% at 5 years. Patients developing ADs were younger and had more nonmalignant diseases (P < .001). ADs target hematopoietic (autoimmune hemolytic anemia, n = 20; Evans syndrome, n = 9; autoimmune thrombocytopenia, n = 11; and immune neutropenia, n = 1) and other tissues (thyroiditis, n = 3; psoriasis, n = 2; Graves disease, n 1; membranous glomerulonephritis, n = 2; rheumatoid arthritis, n = 1; ulcerative colitis, n = 1; and systemic lupus erythematosus, n = 1). Four patients developed 2 ADs (3 cases of immune thrombocytopenia followed by autoimmune hemolytic anemia and 1 Evans syndrome with rheumatoid arthritis). By multivariate analysis, the main risk factor for developing an AD was nonmalignant disease as an indication for CBT (P = .0001). Hematologic ADs were most often treated with steroids, rituximab, and cyclosporine. With a median follow-up of 26 months (range, 2-91), 6 of 52 patients died as a consequence of ADs. We conclude that CBT may be followed by potentially life-threatening, mainly hematologic ADs.
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http://dx.doi.org/10.1182/blood-2012-07-445965DOI Listing
February 2013