Publications by authors named "Mathieu Puyade"

32 Publications

[Autologous hematopoietic cells for severe autoimmune diseases: Guidelines of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC) for immune monitoring and biobanking].

Bull Cancer 2021 Jul 13. Epub 2021 Jul 13.

AP-HP, hôpital Saint-Louis, unité de médecine interne (UF 04): CRMR MATHEC, Maladies auto-immunes et thérapie cellulaire, Centre de Référence des Maladies auto-immunes systémiques Rares d'Ile-de-France MATHEC (FAI2R), 75010 Paris, France; Université de Paris, Institut de recherche Saint-Louis, recherche clinique appliquée à l'hématologie, EA3518, 75010 Paris, France; McGill University, Department of Medicine, H3A 1A1, Montreal, Canada. Electronic address:

Autologous hematopoietic cell transplantation (AHCT) is a new treatment option for patients with severe autoimmune diseases (AD), based on the use of intensive or myeloablative chemotherapy to eradicate the pathogenic autoreactive immune cells and to allow the installation of a new and tolerant immune system during immune reconstitution process. Immune reconstitution analysis after AHCT is required for patients clinical follow-up and to further identify biological and immunological markers of the clinical response to develop individualized AHCT protocols. These MATHEC-SFGM-TC good clinical practice guidelines were developed by a multidisciplinary group of experts including members of the french reference center for stem Cell Therapy in Auto-immune Diseases (MATHEC), hematologists from the French speaking Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC) and experts in immune monitoring and biobanking. The objectives are to provide practical recommandations for immune monitoring and biobanking of samples in patients with AD undergoing AHCT, for routine care purposes and investigational studies.
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http://dx.doi.org/10.1016/j.bulcan.2021.03.020DOI Listing
July 2021

Etiologies of Polyclonal Hypergammaglobulinemia: A scoping review.

Eur J Intern Med 2021 08 11;90:119-121. Epub 2021 Jun 11.

Service de Médecine Interne, Maladies infectieuses et Tropicales, Centre Hospitalier Universitaire (CHU) de Poitiers, 2, rue de la Milétrie, 86021 Poitiers cedex, France.

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http://dx.doi.org/10.1016/j.ejim.2021.05.023DOI Listing
August 2021

Long term outcomes of the French ASTIS systemic sclerosis cohort using the global rank composite score.

Bone Marrow Transplant 2021 Sep 9;56(9):2259-2267. Epub 2021 Jun 9.

Unité de Médecine Interne: Maladies Auto-immunes et Pathologie Vasculaire (UF 04), Centre de Référence des Maladies auto-immunes systémiques Rares d'Ile-de-France MATHEC, AP-HP, Hôpital Saint-Louis, Paris, France.

Two randomised trials (ASTIS, SCOT) of Autologous Hematopoietic Stem Cell Transplantation (AHSCT) versus monthly Cyclophosphamide for severe Systemic Sclerosis (SSc) patients used similar inclusion criteria, but different primary endpoints: event-free-survival (EFS) at 24 months in ASTIS versus the global rank composite score (GRCS) at 54 months in SCOT. Here we analysed the French ASTIS cohort (n = 49) outcome using the same GRCS endpoint as reported in SCOT. All patients, randomised to AHSCT (n = 26) or Cyclophosphamide (n = 23), were evaluated for the non-parametric GRCS endpoint based on: death, EFS, forced vital capacity (FVC), Health Assessment Questionnaire Disability Index (HAQ-DI) and modified Rodnan skin score (mRSS) at 60 months. Secondary endpoints were: EFS, overall survival (OS), HAQ DI and organ status. In intention-to-treat analysis, the GRCS demonstrated superiority for AHSCT (median: 9 versus -19, p = 0.018), mRSS (Δ mRSS: -16 versus -9, p = 0.02), and HAQ-DI (ΔHAQ-DI: -0.89 versus -0.2, p = 0.05) with no significant difference in OS, EFS, lung, heart and kidney function between the groups. In conclusion, this study demonstrates long term benefits of non-myeloablative AHSCT when assessed by the five longitudinal measures within GRCS affording direct primary endpoint comparison between ASTIS and SCOT.
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http://dx.doi.org/10.1038/s41409-021-01355-1DOI Listing
September 2021

Autologous Hematopoietic Stem Cell Transplantation for Behçet's Disease: A Retrospective Survey of Patients Treated in Europe, on Behalf of the Autoimmune Diseases Working Party of the European Society for Blood and Marrow Transplantation.

Front Immunol 2021 6;12:638709. Epub 2021 May 6.

Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, United Kingdom.

Background: Behçet's Disease (BD) is an autoimmune disease mostly presenting with recurrent oral and genital aphthosis, and uveitis. Patients are rarely refractory to immunosuppressive treatments. Autologous hematopoietic stem cell transplantation (aHSCT) is a standard of care in other autoimmune diseases. Some patients with BD have been treated with aHSCT based on compassionate use.

Objectives: Evaluate the outcome of aHSCT in adult patients with BD treated in member centers of the European Society for Blood and Marrow Transplantation (EBMT).

Methods: Adults who received aHSCT primarily for BD were identified retrospectively in the EBMT registry and/or in published literature. Data were extracted from either medical records of the patient or from publications.

Results: Eight out of 9 cases reported to the registry and extracted data of 2 further patients from literature were analyzed. Four were female, median age at onset of BD was 24y (range 9-50). Median age at aHSCT was 32y (27-51). Patients had received median 4 (2-11) previous lines of therapy (89% corticosteroids, 50% methotrexate, anti-TNFα therapy or cyclophosphamide). All patients had active disease before mobilization. Conditioning regimen was heterogeneous. Median follow-up was 48 months (range 6-240). No treatment-related mortality was reported. This procedure induced complete remission (CR) in 80%, partial remission in 10% and lack of response in 10% of the patients. Relapse rate was 30% (2 relapses in patients in CR and 1 relapse in the patient in PR) with panuveitis (n=1), aphthosis (n=2) and arthralgia (n=1). Six patients were in CR. No late complications were reported.

Conclusion: aHSCT has an acceptable safety profile and represents a feasible and relatively effective procedure in severe and conventional treatment-resistant cases of BD and has the potential to stabilize BD in patients with life-threatening involvements.
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http://dx.doi.org/10.3389/fimmu.2021.638709DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8136432PMC
May 2021

Myeloproliferative neoplasms and clonal hematopoiesis in patients with giant cell arteritis: a case-control and exploratory study.

Rheumatology (Oxford) 2021 Apr 9. Epub 2021 Apr 9.

Department of Internal Medicine, Cochin Hospital, National Referral Center for Rare Systemic Autoimmune Diseases, Paris University, Paris, France.

Objectives: Giant cell arteritis (GCA) is a large vessel vasculitis for which triggering factors remain unknown. Clonal hematopoiesis (CH) was associated with atherosclerosis through the induction of inflammation in myeloid cells, and data suggest that CH expansion and inflammation may support each other to induce a proinflammatory loop. Our objective was to describe the impact of JAK2p.V617F-mutated myeloproliferative neoplasms (MPN) on GCA and to screen MPN-free patients for CH mutations.

Methods: We performed a retrospective case-control study comparing characteristics of 21 GCA patients with MPN and 42 age and gender-matched GCA patients without MPN. Also, 18 GCA patients were screened for CH through Next Generation Sequencing.

Results: The most frequent associated MPN was essential thrombocythemia (ET) (n = 11). Compared to controls, GCA patients with MPN had less frequent cephalic symptoms (71.4 vs. 97.6%, p = 0.004) and higher platelets count at baseline [485 (346-586) vs. 346 [IQR 296-418] x 109/L, p = 0.02). There was no difference between groups for other clinical features. Overall survival was significantly shorter in patients with MPN compared to controls [HR 8.2 (95% CI 1.2-56.6), p = 0.03]. Finally, screening for CH using NGS in 15 GCA patients without MPN revealed CH in 33%.

Conclusion: GCA patients with MPN display higher platelets count and shorter overall survival than controls. This association could not be fortuitous given the possible pathophysiological relationship between the two diseases. CH was found in one third of GCA patients, which may be higher than the expected prevalence for similar age, what should be confirmed in a larger cohort.
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http://dx.doi.org/10.1093/rheumatology/keab337DOI Listing
April 2021

Hematopoietic stem cell transplantation in chronic inflammatory demyelinating polyneuropathy: French experience about four patients, under the behalf of French society for bone marrow transplantation.

J Neurol 2021 Apr 2;268(4):1536-1539. Epub 2021 Mar 2.

Unité de Médecine Interne: Maladies Auto-immunes et Pathologie Vasculaire (UF 04), Centre de Référence des Maladies auto-immunes systémiques Rares d'Ile-de-France MATHEC (FAI2R), Assistance Publique-Hôpitaux de Paris, 75010, Paris, France.

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http://dx.doi.org/10.1007/s00415-021-10452-6DOI Listing
April 2021

Subcutaneous Rituximab-MiniCHOP Compared With Subcutaneous Rituximab-MiniCHOP Plus Lenalidomide in Diffuse Large B-Cell Lymphoma for Patients Age 80 Years or Older.

J Clin Oncol 2021 Apr 14;39(11):1203-1213. Epub 2021 Jan 14.

Department of Hematology, Centre Henri Becquerel, UNIROUEN, University of Normandy, INSERM U1245, Rouen, France.

Purpose: The prognosis of elderly patients with diffuse large B-cell lymphoma (DLBCL) is worse than that of young patients. An attenuated dose of chemotherapy-cyclophosphamide, doxorubicin, vincristine, and prednisone plus rituximab (R-miniCHOP)-is a good compromise between efficacy and safety in very elderly patients. In combination with R-CHOP (R2-CHOP), lenalidomide has an acceptable level of toxicity and may mitigate the negative prognosis of the non-germinal center B-cell-like phenotype. The Lymphoma Study association conducted a multicentric, phase III, open-label, randomized trial to compare R-miniCHOP and R2-miniCHOP.

Patients And Methods: Patients of age 80 years or older with untreated DLBCL were randomly assigned into the R-miniCHOP21 group or the R2-miniCHOP21 group for six cycles and stratified according to CD10 expression and age. The first cycle of rituximab was delivered by IV on D1 after a prephase and then delivered subcutaneously on D1 of cycles 2-6. Lenalidomide was delivered at a dose of 10 mg once daily on D1-D14 of each cycle. The primary end point was overall survival (OS).

Results: A total of 249 patients with new DLBCL were randomly assigned (127 R-miniCHOP and 122 R2-miniCHOP). The median age was 83 years (range, 80-96), and 55% of the patients were classified as non-GCB. The delivered dose for each R-miniCHOP compound was similar in both arms. Over a median follow-up of 25.1 months, the intention-to-treat analysis revealed that R2-miniCHOP did not improve OS (2-year OS 66% in R-miniCHOP and 65.7% in R2-miniCHOP arm, = .98) in the overall population or in the non-GCB population. Grade 3-4 adverse events occurred in 53% of patients with R-miniCHOP and in 81% of patients with R2-miniCHOP.

Conclusion: The addition of lenalidomide to R-miniCHOP does not improve OS. Rituximab delivered subcutaneously was safe in this population.
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http://dx.doi.org/10.1200/JCO.20.02666DOI Listing
April 2021

Safety of immune checkpoint inhibitor rechallenge after discontinuation for grade ≥2 immune-related adverse events in patients with cancer.

J Immunother Cancer 2020 12;8(2)

Pharmacologie clinique et Vigilances, Centre Hospitalier Universitaire de Poitiers, Poitiers, France.

Background: Safety of rechallenge of immune checkpoint inhibitor (ICI) after grade ≥2 immune-related adverse events (irAEs) leading to ICI discontinuation remains unclear.

Methods: All adverse drug reactions involving at least one ICI reported up to December 31, 2019 were extracted from the French pharmacovigilance database. Patients were included if they experienced at least one grade ≥2 irAE resulting in ICI discontinuation, with subsequent ICI rechallenge. The primary outcome was the recurrence of at least one grade ≥2 irAE in these patients after ICI rechallenge.

Results: We included 180 patients: 61.1% were men (median age of 66 years), 43.9% had melanoma and 78.9% were receiving anti-programmed cell death 1. First ICI discontinuation was related to 191 irAEs. After ICI rechallenge, 38.9% of the patients experienced at least one grade ≥2 irAE. Among them, 70.0% experienced the same irAE, 25.7% a distinct irAE, and 4.3% both the same and a distinct irAE. Lower recurrence rates of irAEs were associated with rechallenge with the same ICI treatment (p=0.02) or first endocrine irAEs (p=0.003). Gastrointestinal irAEs were more likely to recur (p=0.007). The median duration from ICI discontinuation to rechallenge and the severity of the initial irAE did not predict recurrent irAEs after ICI rechallenge (p=0.53 and p=0.40, respectively).

Conclusions: In this study, 61.1% of the patients who discontinued ICI treatment for grade ≥2 irAEs experienced no recurrent grade ≥2 irAEs after ICI rechallenge. Although ICI rechallenge appears to be safe under close monitoring, it should always be discussed balancing usefulness of rechallenge, patient comorbidities and risk of recurrence of first irAE(s). Due to inherent bias associated with pharmacovigilance studies, further prospective studies are needed to assess risk factors that may influence patient outcomes after ICI rechallenge.
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http://dx.doi.org/10.1136/jitc-2020-001622DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7768965PMC
December 2020

Rehabilitation Before and After Autologous Haematopoietic Stem Cell Transplantation (AHSCT) for Patients With Multiple Sclerosis (MS): Consensus Guidelines and Recommendations for Best Clinical Practice on Behalf of the Autoimmune Diseases Working Party, Nurses Group, and Patient Advocacy Committee of the European Society for Blood and Marrow Transplantation (EBMT).

Front Neurol 2020 11;11:556141. Epub 2020 Dec 11.

Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, United Kingdom.

Autologous haematopoietic stem cell transplantation (AHSCT) is increasingly used to treat people with multiple sclerosis (MS). Supported by an evolving evidence base, AHSCT can suppress active inflammation in the central nervous system and induce long-term changes in immune cell populations, thereby stabilizing, and, in some cases, reversing disability in carefully selected MS patients. However, AHSCT is an intensive chemotherapy-based procedure associated with intrinsic risks, including profound cytopenia, infection, and organ toxicity, accompanied by an on-going degree of immuno-compromise and general deconditioning, which can be associated with a transient increase in functional impairment in the early stages after transplantation. Although international guidelines and recommendations have been published for clinical and technical aspects of AHSCT in MS, there has been no detailed appraisal of the rehabilitation needed following treatment nor any specific guidelines as to how this is best delivered by hospital and community-based therapists and wider multidisciplinary teams in order to maximize functional recovery and quality of life. These expert consensus guidelines aim to address this unmet need by summarizing the evidence-base for AHSCT in MS and providing recommendations for current rehabilitation practice along with identifying areas for future research and development.
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http://dx.doi.org/10.3389/fneur.2020.556141DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7759663PMC
December 2020

[Crohn's disease and autologous hemapoietic cell transplantation: Guidelines from the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC)].

Bull Cancer 2020 Dec 14;107(12S):S140-S150. Epub 2020 Oct 14.

Centre de référence des maladies auto-immunes systémiques rares d'Île-de-France, hôpital St-Louis (AP-HP), unité de médecine interne : maladies auto-immunes et pathologie vasculaire (UF 04), 1, avenue Claude-Vellefaux, 75010 Paris, France; Université Paris-Denis-Diderot, institut de recherche Saint-Louis, EA 3518, Sorbonne Paris Cité, France; McGill University, department of internal medicine, Montreal, Canada. Electronic address:

Crohn's Disease (CD) is an auto-inflammatory disease, which may involve the entire gastro-intestinal tract. CD is diagnosed on several clinical, biological, endoscopic and histological criteria. First line therapy is based on oral or iv steroids. In case of steroids dependence or resistance, several types of immunosuppressive or immunomodulating therapies are available: classical antimetabolites (thiopurines or methotrexate) or monoclonal antibodies against TNFα, against interleukin 12/23 or against integrin. Nonetheless, Crohn's disease may remain active despite the use of several lines of therapy. In such cases, autologous hematopoietic cell transplantation (AHCT) is an effective therapeutic option in highly selected CD patients with specific criteria. The MATHEC-SFGM-TC Good Clinical Practice Guidelines (GCPG) were developed by a multidisciplinary group of experts including gastroenterologists, hematologists and members of the reference center for stem cell therapy in auto-immune diseases (MATHEC), including members of the French groupe d'étude thérapeutique des affections inflammatoires du tube digestif(GETAID) under the auspices of the French speaking Society of bone marrow transplantation and cellular therapy (SFGM-TC). The aim of the present guidelines is to define the eligibility criteria for CD patients when candidates to AHCT, the procedures for mobilization of hematopoietic stem cell (HSC), conditioning regimen and standardized follow-up after AHCT including monitoring of gastroenterological treatments during AHCT and thereafter throughout all follow-up.
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http://dx.doi.org/10.1016/j.bulcan.2020.08.009DOI Listing
December 2020

Multiple Myeloma: An Overview of the Current and Novel Therapeutic Approaches in 2020.

Cancers (Basel) 2020 Oct 8;12(10). Epub 2020 Oct 8.

Department of Internal Medicine, and CIC1402 INSERM unit, Poitiers University Hospital, 2 Rue de la Milétrie, 86021 Poitiers, France.

The survival rate of multiple myeloma (MM) patients has drastically increased recently as a result of the wide treatment options now available. Younger patients truly benefit from these innovations as they can support more intensive treatment, such as autologous stem cell transplant or multiple drug association (triplet, quadruplet). The emergence of immunotherapy allowed new combinations principally based on monoclonal anti-CD38 antibodies for these patients. Still, the optimal induction treatment has not been found yet. While consolidation is still debated, maintenance treatment is now well acknowledged to prolong survival. Lenalidomide monotherapy is the only drug approved in that setting, but many innovations are expected. Older patients, now logically named not transplant-eligible, also took advantage of these breakthrough innovations as most of the recent drugs have a more acceptable safety profile than previous cytotoxic agents. For this heterogenous subgroup, geriatric assessment has become an essential tool to identify frail patients and provide tailored strategies. At relapse, options are now numerous, especially for patients who were not treated with lenalidomide, or not refractory at least. Concerning lenalidomide refractory patients, approved combinations are lacking, but many trials are ongoing to fill that space. Moreover, innovative therapeutics are increasingly being developed with modern immunotherapy, such as chimeric antigen receptor T-cells (CAR-T cells), bispecific antibodies, or antibody-drug conjugates. For now, these treatments are usually reserved to heavily pre-treated patients with a poor outcome. MM drug classes have tremendously extended from historical alkylating agents to current dominant associations with proteasome inhibitors, immunomodulatory agents, and monoclonal anti-CD38/anti SLAMF7 antibodies. Plus, in only a couple of years, several new classes will enter the MM armamentarium, such as cereblon E3 ligase modulators (CELMoDs), selective inhibitors of nuclear export, and peptide-drug conjugates. Among the questions that will need to be answered in the years to come is the position of these new treatments in the therapeutic strategy, as well as the role of minimal residual disease-driven strategies which will be a key issue to elucidate. Through this review, we chose to enumerate and comment on the most recent advances in MM therapeutics which have undergone major transformations over the past decade.
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http://dx.doi.org/10.3390/cancers12102885DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7600346PMC
October 2020

Bone marrow biopsy diagnostic yield in internal medicine.

Postgrad Med 2021 Jan 4;133(1):89-95. Epub 2020 Nov 4.

Service de Médecine Interne, Maladies Infectieuses et Tropicales, Centre Hospitalier Universitaire de Poitiers , Poitiers, France.

Background: Trephine bone marrow biopsy (BMB) in internal medicine has only been studied in fever of unknown origin and inflammation of unknown origin. The aim was to assess BMB diagnostic yield according to main indications and patient characteristics in internal medicine. Quality of BMB and contribution of bone marrow aspiration (BMA) to BMB were also analyzed.

Methods: BMB performed in the internal medicine department of Poitiers university hospital between January 2000 and December 2015 were retrospectively analyzed. Patient characteristics, BMB indications, quality parameters, and results were collected from medical records. Contributive BMB was BMB allowing accurate final diagnosis. Diagnostic yield was the proportion of contributive BMB among total BMB performed.

Results: A total of 468 BMBs conducted for primary diagnostic purpose from 468 patients were analyzed. Cytopenia(s) and the indication 'adenopathy and/or splenomegaly and/or hepatomegaly' represented 70% of the indications. Overall BMB diagnostic yield was 32.7%, lymphoma being the main histologic finding (31%). Among indications, cytopenia(s) had the highest diagnostic yield (49.1%). Isolated fever of unknown origin had low diagnostic yield (5.6%). Factors independently associated with contributive BMB were: anemia, neutropenia, circulating immature granulocytes or blasts, monoclonal gammopathy, period of BMB processing, quality of BMB, and immunohistochemestry (IHC) analysis. Concomitant BMA improved diagnostic yield by 5.5%, mostly for myelodysplastic syndromes.

Conclusion: Cytopenia(s), blood cythemias and monoclonal gammopathy are indications with the highest diagnostic yield. Concomitant BMA and IHC analysis should be systematically performed to increase BMB diagnostic yield in internal medicine.
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http://dx.doi.org/10.1080/00325481.2020.1835118DOI Listing
January 2021

Association of Autologous Hematopoietic Stem Cell Transplantation in Systemic Sclerosis With Marked Improvement in Health-Related Quality of Life.

Arthritis Rheumatol 2021 02 29;73(2):305-314. Epub 2020 Dec 29.

Centre de Référence des Maladies Auto-Immunes Systémiques Rares d'Ile-de-France, AP-HP, Hôpital St. Louis, Université de Paris, Institut de Recherche St. Louis, EA 3518, Hôpital St. Antoine, Paris, France, and McGill University, Montreal, Quebec, Canada.

Objective: To quantify the magnitude, domains, and duration of change in health-related quality of life (HRQoL) in patients with systemic sclerosis (SSc) who underwent autologous hematopoietic stem cell transplantation (HSCT) as compared to SSc patients with similar characteristics who did not undergo autologous HSCT.

Methods: The study was designed as a retrospective study comparing SSc patients who underwent autologous HSCT and SSc patients who met the criteria for transplantation but were treated with conventional care. Outcomes included scores on the 36-item Short Form (SF-36) health survey and the Health Assessment Questionnaire (HAQ) and its disease-specific symptom scales. Differences in scores between the groups were compared using linear models, adjusting for baseline scores and inverse probability of treatment and censoring weights.

Results: In total, 41 SSc patients who underwent autologous HSCT and 65 SSc patients treated with conventional care were compared. In marginal linear weighted models, the SF-36 physical component summary score was a mean ± SEM 7.02 ± 1.94 points higher at the first annual visit (P = 0.001) and 14.40 ± 6.16 points higher at the seventh annual visit (P = 0.03) in patients treated with autologous HSCT compared to the conventional care group. HAQ scores were significantly better in the autologous HSCT group compared to the conventional care group during follow-up (mean ± SEM difference from baseline -0.57 ± 0.13 [P < 0.001] at the first annual visit and -0.94 ± 0.49 [P = 0.07] at the seventh annual visit). There were no differences in the SF-36 mental component summary scores between the 2 groups either at baseline or during follow-up.

Conclusion: This study provides robust complementary HRQoL data, including overall and event-free survival data, to expand on the standard repertoire of biomedical variables, thus potentially supporting the physical benefits of autologous HSCT in patients with SSc.
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http://dx.doi.org/10.1002/art.41519DOI Listing
February 2021

How should we diagnose and treat blastic plasmacytoid dendritic cell neoplasm patients?

Blood Adv 2019 12;3(24):4238-4251

Laboratoire d'Hématologie, CHU Estaing 1, Clermont-Ferrand, France.

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and aggressive leukemia for which we developed a nationwide network to collect data from new cases diagnosed in France. In a retrospective, observational study of 86 patients (2000-2013), we described clinical and biological data focusing on morphologies and immunophenotype. We found expression of markers associated with plasmacytoid dendritic cell origin (HLA-DRhigh, CD303+, CD304+, and cTCL1+) plus CD4 and CD56 and frequent expression of isolated markers from the myeloid, B-, and T-lymphoid lineages, whereas specific markers (myeloperoxidase, CD14, cCD3, CD19, and cCD22) were not expressed. Fifty-one percent of cytogenetic abnormalities impact chromosomes 13, 12, 9, and 15. Myelemia was associated with an adverse prognosis. We categorized chemotherapeutic regimens into 5 groups: acute myeloid leukemia (AML)-like, acute lymphoid leukemia (ALL)-like, lymphoma (cyclophosphamide, doxorubicin, vincristine, and prednisone [CHOP])-like, high-dose methotrexate with asparaginase (Aspa-MTX) chemotherapies, and not otherwise specified (NOS) treatments. Thirty patients received allogeneic hematopoietic cell transplantation (allo-HCT), and 4 patients received autologous hematopoietic cell transplantation. There was no difference in survival between patients receiving AML-like, ALL-like, or Aspa-MTX regimens; survival was longer in patients who received AML-like, ALL-like, or Aspa-MTX regimens than in those who received CHOP-like regimens or NOS. Eleven patients are in persistent complete remission after allo-HCT with a median survival of 49 months vs 8 for other patients. Our series confirms a high response rate with a lower toxicity profile with the Aspa-MTX regimen, offering the best chance of access to hematopoietic cell transplantation and a possible cure.
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http://dx.doi.org/10.1182/bloodadvances.2019000647DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6929390PMC
December 2019

[Indication of autologous stem cell transplantation in chronic inflammatory demyelinating polyneuropathy: Guidelines from the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC)].

Bull Cancer 2020 Jan 7;107(1S):S104-S113. Epub 2019 Dec 7.

AP-HP, hôpital St-Louis, centre de référence des maladies auto-immunes systémiques rares d'Île-de-France, unité de médecine interne : maladies auto-immunes et pathologie vasculaire (UF 04), 1, avenue Claude Vellefaux, 75010 Paris, France; Institut de recherche Saint-Louis, université Paris Denis Diderot, Sorbonne Paris Cité, EA 3518, Paris, France; Department of Internal Medicine, McGill University, Montreal, Canada. Electronic address:

Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) is a chronic autoimmune disease involving the peripheral nervous system, characterized by focal and segmental demyelination accounting for neurological deficit. CIDP diagnosis is based on several criteria and requires the presence of specific clinical symptoms and of demyelinating criteria on the electroneuromyogram (ENMG) or of additional supportive criteria (spinal fluid examination with dissociation between albumin level and cellular abnormalities, nervous abnormalities on MRI or other minor abnormalities on ENMG, demyelinating features on nerve biopsy or patient improvement under so-called first-line therapy with immunodulator treatment). After failure of two successive first line immunomodulating drug therapies (corticosteroids, immunomodulating immunoglobulins, or plasma exchange), several options can be considered as second line therapies. The efficacy of autologous hematopoietic cell transplantation (AHCT) has been shown in CIDP patients. The aim of these recommendations established by a working group of experts from the "Société française de greffe de moelle osseuse et thérapie cellulaire (SFGM-TC)", the group "maladies auto-immunes et thérapie cellulaire (MATHEC)" and the "filière de santé maladies rares neuromusculaire (FILNEMUS)" is to specify the eligibility criteria for AHCT in CIPD patients, to describe the mobilization and the conditioning regimen for the AHCT procedure, as well as the patient standardized post-transplant follow-up and the management of neurological treatment throughout the all procedure.
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http://dx.doi.org/10.1016/j.bulcan.2019.11.002DOI Listing
January 2020

Health-related quality of life in systemic sclerosis before and after autologous haematopoietic stem cell transplant-a systematic review.

Rheumatology (Oxford) 2020 04;59(4):779-789

Unité de Médecine Interne: Maladies Auto-immunes et Pathologie Vasculaire (UF 04), Centre de Référence des Maladies auto-immunes systémiques Rares d'Ile-de-France, AP-HP, Hôpital St-Louis, Saint Louis.

Objectives: In severe rapidly progressive SSc, autologous haematopoietic stem cell transplantation (AHSCT) allows significant improvements in overall and event-free survival. We undertook this study to identify, appraise and synthesize the evidence on health-related quality of life (HRQoL) before and after AHSCT for SSc.

Methods: We performed a systematic review of the literature, following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines, in PubMed and ScienceDirect from database inception to 1 February 2019. All articles with original HRQoL data were selected.

Results: The search identified 1080 articles, of which 8 were selected: 3 unblinded randomized controlled trials [American Scleroderma Stem Cell versus Immune Suppression Trial (ASSIST), Autologous Stem Cell Transplantation International Scleroderma, Scleroderma: Cyclophosphamide or Transplantation), 3 uncontrolled phase I or II trials and 2 cohort studies. HRQoL data from 289 SSc patients treated with AHSCT and 125 treated with intravenous CYC as a comparator with median 1.25-4.5 years follow-up were included. HRQoL was evaluated with the HAQ Disability Index (HAQ-DI; 275 patients), the 36-item Short Form Health Survey (SF-36; 249 patients) and the European Quality of Life 5-Dimensions questionnaire (EQ-5D; 138 patients). The quality of the studies was moderate to low. AHSCT was associated with significant improvement in the HAQ-DI (P = 0.02-<0.001), SF-36 Physical Component Summary score (P = 0.02-<0.0001) and EQ-5D index-based utility score (P < 0.001). The SF-36 Mental Component Summary score improved in the ASSIST (n = 19) and one small retrospective cohort (n = 30 patients, P = 0.005) but did not improve significantly in 2 randomized controlled trials (n = 200 patients, P = 0.1-0.91).

Conclusion: AHSCT in severe SSc patients is associated with significant and durable improvement in physical HRQoL.
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http://dx.doi.org/10.1093/rheumatology/kez300DOI Listing
April 2020

Perspectives on Scedosporium species and Lomentospora prolificans in lung transplantation: Results of an international practice survey from ESCMID fungal infection study group and study group for infections in compromised hosts, and European Confederation of Medical Mycology.

Transpl Infect Dis 2019 Oct 22;21(5):e13141. Epub 2019 Jul 22.

Université de Paris, APHP, Service des Maladies Infectieuses et Tropicales, Hôpital Necker-Enfants Malades, Centre d'Infectiologie Necker-Pasteur, Institut Imagine, Paris, France.

Background: Scedosporium species and Lomentospora prolificans (S/L) are the second most common causes of invasive mold infections following Aspergillus in lung transplant recipients.

Methods: We assessed the current practices on management of S/L colonization/infection of the lower respiratory tract before and after lung transplantation in a large number of lung transplant centers through an international practice survey from October 2016 to March 2017.

Results: A total of 51 respondents from 45 lung transplant centers (17 countries, 4 continents) answered the survey (response rate 58%). S/L colonization was estimated to be detected in candidates by 48% of centers. Only 18% of the centers used a specific medium to detect S/L colonization. Scedosporium spp. colonization was a contraindication to transplantation in 10% of centers whereas L prolificans was a contraindication in 31%; 22% of centers declared having had 1-5 recipients infected with S/L in the past 5 years.

Conclusions: This survey gives an overview of the current practices regarding S/L colonization and infection in lung transplant centers worldwide and underscores the need of S/L culture procedure standardization before implementing prospective studies.
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http://dx.doi.org/10.1111/tid.13141DOI Listing
October 2019

Large-vessel vasculitis diagnosed between 50 and 60 years: Case-control study based on 183 cases and 183 controls aged over 60 years.

Autoimmun Rev 2019 Jul 4;18(7):714-720. Epub 2019 May 4.

Department of Internal Medicine, Hôpital Lariboisière, Paris, France.

Background: Age at onset of large-vessel vasculitis (LVV) is commonly used to distinguish giant cell arteritis (GCA) and Takayasu arteritis (TA). However, LVV between age 50 and 60 years may be difficult to classify.

Methods: We conducted a retrospective study including LVV aged between 50 and 60 years at onset (LVV, cases) and compared them to LVV aged over 60 years (LVV, controls). LVV was defined histologically and/or morphologically. Controls fulfilled ACR 1990 criteria for GCA or presented isolated aortitis.

Results: We included 183 LVV and 183 gender-matched LVV. LVV had more frequent peripheral limb manifestations (23 vs. 5%), and less frequent cephalic (73 vs. 90%) and ocular signs (17 vs. 27%) than LVV. Compared to LVV, CT angiography and PET/CT scan were more frequently abnormal in LVV (74 vs. 38%, and 90 vs. 72%, respectively), with aorta being more frequently involved (78 vs. 47%). By multivariate analysis, absence of cephalic symptoms, presence of peripheral limb ischemia and aorta involvement, and increased CRP level were significantly associated with LVV presentation compared to LVV. At last follow-up, compared to LVV, LVV received significantly more lines of treatment (2 vs. 1), more frequent biologics (12 vs. 3%), had more surgery (10 vs. 0%), and had higher prednisone dose (8.8 vs. 6.5 mg/d) at last follow-up, CONCLUSION: LVV onset between 50 and 60 years identifies a subset of patients with more frequent aorta and peripheral vascular involvement and more refractory disease compared to patients with LVV onset after 60.
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http://dx.doi.org/10.1016/j.autrev.2019.05.008DOI Listing
July 2019

Prognosis of Streptococcus pneumoniae endocarditis in France, a multicenter observational study (2000-2015).

Int J Cardiol 2019 08 17;288:102-106. Epub 2019 Apr 17.

Université de Poitiers, Poitiers, France; CHU de Poitiers, Service de maladies infectieuses et tropicales, Poitiers, France; Inserm U1070, Poitiers, France. Electronic address:

Background: Streptococcus pneumoniae is responsible for <2% of infective endocarditis (IE). The aim of this study was to assess the prognosis of pneumococcal IE.

Methods: This multicentric observational retrospective study included adult patients presenting with definite S. pneumoniae IE according to modified Dukes criteria from four French university hospitals over a 15-year period, January 2000-December 2015. Survival rate at 90 days and 2 years after diagnosis, appropriateness of antibiotherapy, and pneumococcal vaccination status were determined. Risk factors for mortality were studied by univariate analysis.

Results: Of 3886 patients admitted with IE during the study period, 50 (1.3%) had pneumococcal IE, mostly males (n = 38, 76%), with a mean age of 60 ± 14 years. Predisposing conditions for IE or for invasive pneumococcal disease (IPD) involved 24% and 78% of the cases, respectively. Only 2 patients were vaccinated against pneumococcus before IE and 13 (26%) after IE. Antimicrobial strategy was in accordance with the 2015 ESC Guidelines in 28%. Cardiac surgery was performed in 56%, and was associated with better survival (p = 0.012). In the 40 patients followed until 2 years, the survival rate was 67%, deaths occurring mostly before 90 days. Age ≥ 65 was a risk factor for mortality (p = 0.011).

Conclusion: Pneumococcal IE remains rare but with a poor prognosis. Resort to surgery is yet to be determined. Predisposing conditions for IPD are the main factors leading to pneumococcal IE. They could be prevented by vaccine coverage improvement.
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http://dx.doi.org/10.1016/j.ijcard.2019.04.048DOI Listing
August 2019

[Prophylaxis of infections post-allogeneic transplantation: Guidelines from the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC)].

Bull Cancer 2019 Jan 4;106(1S):S23-S34. Epub 2019 Jan 4.

Institut d'hématologie de Basse-Normandie, centre hospitalier universitaire, avenue de la Côte-de-Nacre, 14000 Caen, France. Electronic address:

Allogeneic hematopoietic stem cell transplantation is a curative treatment for many hematological diseases. However, this procedure causes the patient to be susceptible to infection. Prophylactic treatments are administered in clinical practice even thought the level of evidence of their effectiveness is not always high. In addition, changes in the transplantation procedures - use of reduced intensity conditioning, development of alternative graft sources - must lead to a rethinking of attitudes towards prophylaxis. Our working group based its recommendations on a review of referential articles and publications on the subject found in the literature. These recommendations concern the prophylaxis of infections caused by HSV1, HSV2, varicella zoster, and hepatitis B, as well as anti-bacterial and digestive decontamination prophylaxis, prevention of pneumocystis, toxoplasmosis, tuberculosis, as well as prophylaxis of fungal infections. Other infectious agents usually involved in infections post-allotransplant have been the subject of another set of recommendations from the French Society of Bone Marrow Transplantation and Cellular Therapy.
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http://dx.doi.org/10.1016/j.bulcan.2018.08.017DOI Listing
January 2019

[Indications and follow-up for autologous hematopoietic stem cell transplantation in multiple sclerosis: Guidelines from the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC) in association with the Francophone Society of Multiple Sclerosis].

Bull Cancer 2019 Jan 5;106(1S):S92-S101. Epub 2018 Dec 5.

Hôpital Saint-Louis, centre de référence des maladies auto-immunes systémiques rares d'Île-de-France, filière FAI2R, IUH EA-3518, UF04, unité de médecine interne, maladies auto-immunes et pathologie vasculaire, 1, avenue Claude-Vellefaux, 75475 Paris, France. Electronic address:

The Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC) organized the 8th allogeneic hematopoietic stem cell transplantation clinical practices harmonization workshop series in September 2017 in Lille, France. In this article we give the indications of autologous stem cell transplantation in multiple sclerosis as well as recommendations regarding post-transplant follow-up of patients under the hospice of the SFGM-TC and the Francophone Society of Multiple Sclerosis.
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http://dx.doi.org/10.1016/j.bulcan.2018.11.002DOI Listing
January 2019

Reproducibility and Utility of the 6-minute Walk Test in Systemic Sclerosis.

J Rheumatol 2018 08 1;45(9):1273-1280. Epub 2018 Jul 1.

From the Service de Médecine Interne, CHU Toulouse Purpan; Faculté de Médecine de Toulouse, Toulouse; Hématologie clinique et thérapie cellulaire, and Unité de Médecine Interne, Maladies Auto-immunes et Pathologie Vasculaire, UF 04, Centre de référence des maladies auto-immunes systémiques rares d'Ile-de-France, Filière (FAI2R), AP-HP, hôpital Saint-Antoine; Université Paris Diderot, Sorbonne Paris Cité, Paris, France; Service de médecine interne, Centre national de référence "Lupus, syndrome des antiphospholipides et autres maladies auto-immunes systémiques rares," Hôpital Pierre Zobda Quitman, CHU de Martinique, Fort de France, Martinique; Service de Médecine Interne et Maladies Infectieuses, CHU de Poitiers, Poitiers, France; Department of Cardiology, Department of Internal Medicine, and Department of Rheumatology, Ghent University Hospital, Ghent, Belgium.

Objective: To assess the reproducibility and the utility of the 6-minute walk test (6MWT) in systemic sclerosis (SSc).

Methods: All patients with SSc who underwent at least two 6MWT within a minimum 3-month interval plus simultaneous routine clinical, biological, and functional evaluations were consecutively enrolled in this observational study over 6 years. Following American Thoracic Society guidelines, each 6MWT was repeated twice to assess the 6-minute walk distance (6MWD) reproducibility, with the highest value being reported for subsequent analysis.

Results: Among 56 (38 female) included patients aged 46 ± SD 12.7 years, with 17 ± 10 modified Rodnan skin score (mRSS) and 1 ± 0.8 Scleroderma Health Assessment Questionnaire (SHAQ) at first referral, 277 6MWT evaluations (5 ± 3.9 6MWT per patient) were performed over 23 ± 22.5 months followup. Meanwhile, 8 deaths (87.5% SSc-related) occurred. The mean 6MWD absolute value was 457 ± 117 m with a 4 ± 2.2 mean Borg dyspnea score. The 6MWD intraclass correlation coefficient was 0.996 (95% CI 0.995-0.999, p < 0.0001). In multivariate linear regression analysis, these factors were independently associated with a lower 6MWD: sex (R = 0.47, p < 0.0001), mRSS (R = 0.47, p = 0.008), tendon friction rub (R = 0.47, p = 0.003), SHAQ (R = 0.47, p = 0.02), muscle disability score (R = 0.47, p = 0.03), DLCO% (R = 0.47, p = 0.0008), and left ventricular ejection fraction (R = 0.47, p = 0.006). The 6MWD at first referral was an independent predictor for the overall mortality (HR 0.99, 95% CI 0.988-0.999) and the SSc-related mortality (HR 0.99, 95% CI 0.988-0.999).

Conclusion: We show strong reproducibility for the 6MWD and confirm the 6MWT utility to assess the overall prognosis of patients with SSc.
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http://dx.doi.org/10.3899/jrheum.170994DOI Listing
August 2018

Efficacy of etoposide for myelodysplasia cutis.

Eur J Dermatol 2018 04;28(2):246-247

Service de Dermatologie, Centre Hospitalier Universitaire de Poitiers, Poitiers, France.

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http://dx.doi.org/10.1684/ejd.2018.3225DOI Listing
April 2018

Pregnancy and myeloproliferative neoplasms : A retrospective monocentric cohort.

Obstet Med 2017 Dec 4;10(4):165-169. Epub 2017 Aug 4.

CHU de Poitiers, Obstetric Medicine Clinic, Service de Réanimation Médicale et de Médecine Interne, Poitiers, France.

Background: The most frequent myeloproliferative neoplasms are essential thrombocythemia and chronic myelogenous leukemia, which usually manifests with thrombocytosis. Only essential thrombocythemia is associated with morbidity during pregnancy (recurrent miscarriages, intrauterine fetal death, small for gestational age and preeclampsia). The aim of this paper is to describe outcomes of pregnancy in women with myeloproliferative neoplasms seen at a single academic institution.

Methods: Data were collected retrospectively from 2002 to 2015. Descriptive analyses were performed.

Results: Eighteen pregnancies in 13 patients and 17 births were identified. One patient had recurrent miscarriages. There were two intrauterine fetal deaths, three small for gestational age linked to vascular placenta pathology and one preeclampsia. All of these mothers harbored JAK2V617F mutation. Two out of three patients with small for gestational age developed a venous thrombosis in the two years following delivery.

Conclusion: Thrombocytosis associated with myeloproliferative neoplasms should be considered as a risk factor for maternal and fetal complications.
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http://dx.doi.org/10.1177/1753495X17708896DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5714108PMC
December 2017

[Indications and follow-up for autologous hematopoietic stem cell transplantation in autoimmune and autoinflammatory diseases: Guidelines from the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC)].

Bull Cancer 2017 Dec 22;104(12S):S169-S180. Epub 2017 Nov 22.

Hôpital Saint-Louis, UF04, unité de médecine interne, maladies auto-immunes et pathologie vasculaire, centre de référence des maladies auto-immunes systémiques rares d'Ile-de-France, Filière 'FAI2R', 1, avenue Claude-Vellefaux, 75475 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 2017 in Lille, France and updated recommendations for indications and follow-up in autologous hematopoietic stem cell transplantation in autoimmune and autoinflammatory diseases, previously published under the auspices of SFGM-TC.
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http://dx.doi.org/10.1016/j.bulcan.2017.06.019DOI Listing
December 2017

Should We Screen for Janus Kinase 2 V617F Mutation in Cerebral Venous Thrombosis?

Cerebrovasc Dis 2017 14;44(3-4):97-104. Epub 2017 Jun 14.

Department of Neurology, S. Giovanni Calibita-Fatebenefratelli Hospital, Rome, Italy.

Background: The presence of Janus Kinase 2 (JAK2) V617F mutation represents a major diagnostic criterion for detecting myeloproliferative neoplasms (MPN) and even in the absence of overt MPN, JAK2 V617F mutation is associated with splanchnic vein thrombosis. However, the actual prevalence and diagnostic value of the JAK2 V617F mutation in patients with cerebral venous thrombosis (CVT) are not known. The aims of this study were to assess the prevalence of JAK2 V617F mutation in a large group of consecutive CVT patients, to detect clinical, biological, and radiological features associated with the mutation, and to determine the long-term venous thrombosis recurrence rate in CVT patients with JAK2 mutation but without overt MPN in order to recommend the best preventive treatment.

Methods: This was a prospective study conducted on consecutive patients with a first-ever radiologically confirmed CVT. JAK2 V617F mutation analysis was assessed in all the study subjects. JAK2 V617F-positive patients were followed up to detect new venous thrombotic events.

Results: Of the 125 included subjects, 7 were found to have JAK2 V617F mutation (5.6%; 95% CI 2.3-11.2). Older age (p = 0.039) and higher platelet count (p = 0.004) were independently associated with JAK2 V617F positivity in patients without overt MPN. During a mean follow-up period of 59 (SD 46) months, 2 JAK2 V617F-positive patients presented with 4 new venous thromboembolic events.

Conclusions: Screening for the JAK2 V617F mutation in CVT patients seems to be useful even in the absence of overt MPN and/or in the presence of other risk factors for CVT because of its relatively high prevalence and the risk of thrombosis recurrence.
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http://dx.doi.org/10.1159/000471891DOI Listing
June 2018

Age-related health care disparities in multiple myeloma.

Hematol Oncol 2018 Feb 21;36(1):224-231. Epub 2017 Apr 21.

INSERM CIC 1402, CHU, Poitiers, France.

Age is a well-known factor in solid tumours linked to lower adherence to guidelines. Scarce data exist for haematologic malignancies such as multiple myeloma (MM). The aim of the study was to investigate the relationships among age, adherence to guidelines in MM, and overall survival (OS).The Poitou-Charentes cancer registry has exhaustively registered incident cases of MM from 2008 to 2010. Diagnosis, staging, prognosis, and first-line treatment were compared to the international guidelines. Three hundred and sixty-seven patients aged 36 to 93 years were included. Compliance to diagnostic procedure was 98%, staging 62%, prognosis 30%, and first-line treatment 89%. Cytogenetic analysis was compliant in 37%. Younger age was the strongest factor associated with compliant provision of care (odds ratio 14.4 [6.1-33.8] for <66 years and 2.3 [0.9-6.1] for 66-74 years; P < .0001). The second independent factor was active versus smouldering myeloma (odds ratio 3.5 [1.6-7.3]; P = .0009). Adherence to guidelines is related with OS in multivariate analysis hazard ratio: 1.6 [1.0-2.5]; P = .03. Age is linked with inadequate provision of care in myeloma, particularly prognosis and first-line treatment. Compliance to guidelines seems to be related to OS taking into account the main prognostic factors. Future guidelines should stress the point that age and frailty need to be taken into account in myeloma care.
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http://dx.doi.org/10.1002/hon.2422DOI Listing
February 2018

Ibrutinib Is Effective in the Treatment of Autoimmune Haemolytic Anaemia in Mantle Cell Lymphoma.

Case Rep Oncol 2017 Jan-Apr;10(1):127-129. Epub 2017 Jan 27.

CHU de Poitiers, Service d'Onco-Hématologie et Thérapie Cellulaire, Poitiers, France.

Autoimmune haemolytic anaemia (AIHA) in mantle cell lymphoma (MCL) is a rare but life-threatening complication. To date, there are no relevant data for treatment of AIHA in MCL. Ibrutinib, which has been approved for relapse/refractory MCL, is an immunomodulatory drug inhibiting Th2 activation and consequently the production of autoantibodies. We report a case of MCL with AIHA in which this form of anaemia was not controlled with the usual chemotherapy. Ibrutinib was used when MCL with AIHA relapsed, and it allowed rapid remission of AIHA and rapid discontinuation of steroid therapy.
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http://dx.doi.org/10.1159/000456002DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5301091PMC
January 2017

VTD is superior to VCD prior to intensive therapy in multiple myeloma: results of the prospective IFM2013-04 trial.

Blood 2016 05 21;127(21):2569-74. Epub 2016 Mar 21.

University Hospital, Metz, France;

The Intergroupe Francophone du Myélome conducted a randomized trial to compare bortezomib-thalidomide-dexamethasone (VTD) with bortezomib-cyclophosphamide-dexamethasone (VCD) as induction before high-dose therapy and autologous stem cell transplantation (ASCT) in patients with newly diagnosed multiple myeloma. Overall, a total of 340 patients were centrally randomly assigned to receive VTD or VCD. After 4 cycles, on an intent-to-treat basis, 66.3% of the patients in the VTD arm achieved at least a very good partial response (primary end point) vs 56.2% in the VCD arm (P = .05). In addition, the overall response rate was significantly higher in the VTD arm (92.3% vs 83.4% in the VCD arm; P = .01). Hematologic toxicity was higher in the VCD arm, with significantly increased rates of grade 3 and 4 anemia, thrombocytopenia, and neutropenia. On the other hand, the rate of peripheral neuropathy (PN) was significantly higher in the VTD arm. With the exception of hematologic adverse events and PN, other grade 3 or 4 toxicities were rare, with no significant differences between the VTD and VCD arms. Our data support the preferential use of VTD rather than VCD in preparation for ASCT. This trial was registered at www.clinicaltrials.gov as #NCT01564537 and at EudraCT as #2013-003174-27.
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http://dx.doi.org/10.1182/blood-2016-01-693580DOI Listing
May 2016
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