Search Our Scientific Publications & Authors

Biol Blood Marrow Transplant 2019 Feb 19. Epub 2019 Feb 19.

Medical College of Wisconsin, Milwaukee, WI.

Purpose: The curative potential of autologous hematopoietic cell transplantation (autoHCT) for male germ cell tumors (GCT) is well established. Optimal timing and number (single, ST vs. tandem, TT vs. Read More

Biol Blood Marrow Transplant 2019 Feb 19. Epub 2019 Feb 19.

Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington.

Multiple sclerosis (MS) is a chronic, disabling, immune-mediated, central nervous system demyelinating and degenerative disease. Approved disease modifying therapies may be incompletely effective in some patients with highly active relapsing disease and high risk of disability. Immunoablative or myeloablative therapy followed by autologous hematopoietic cell transplantation (AHCT) has been investigated in retrospective studies, clinical trials, and meta-analyses/systematic reviews as an approach to address this unmet clinical need. Read More

Biol Blood Marrow Transplant 2019 Feb 19. Epub 2019 Feb 19.

Department of Oncology and Hematology, Shimane University Hospital, Izumo, Japan. Electronic address:

We analyzed data of 64,539 consecutive patients in the national Japanese transplant registry, including 40,195 after allogeneic stem cell transplantation, 24,215 after autologous stem cell transplantation, and 129 after syngeneic stem cell transplantation, and 299 developed Epstein-Barr virus positive post-transplant lymphoproliferative disorder. The probability at 2 years was 0.79% after allogeneic transplantation, 0. Read More

PLoS One 2019 22;14(2):e0212708. Epub 2019 Feb 22.

Haematology Department, Hospital Regional Universitario de Málaga, Instituto de Investigación Biomédica de Málaga (IBIMA), Málaga, Spain.

Background: The analysis of molecular haematopoietic chimerisms (HC) has become a well-established method to monitor the transplant evolution and to assess the risk of relapse after allogeneic stem cells transplantation (allo-STC). Different techniques and molecular markers are being used for chimerism surveillance after transplantation, including quantitative real-time PCR (qPCR) and the recently developed digital PCR (dPCR). This study aims to compare the sensitivity and accuracy of both methods to quantify HC and predict early relapse. Read More

Hematol Transfus Cell Ther 2019 Jan-Mar;41(1):69-75. Epub 2018 Jul 29.

Fundação Doutor Amaral Carvalho, Jaú, SP, Brazil.

Graft-versus-host disease is one of the main causes of morbidity and mortality in patients submitted to hematopoietic stem cell transplantation. This study reviewed the prevalence of lower female genital tract graft-versus-host disease following hematopoietic stem cell transplantation. A systematic search of the literature for articles published between 1982 and 2015 was performed. Read More

Hematol Transfus Cell Ther 2019 Jan-Mar;41(1):1-6. Epub 2018 Jul 10.

Hospital das Clínicas da Universidade Federal de Minas Gerais (HC UFMG), Belo Horizonte, MG, Brazil.

Background: ABO blood group incompatibility between donor and recipient is associated with a number of immunohematological complications, but is not considered a major contraindication to allogeneic hematopoietic stem cell transplantation. However, available evidence from the literature seems to be conflicting as to the impact of incompatibility on overall survival, event-free survival, transplant-related mortality, graft-versus-host disease, and time to neutrophil and platelet engraftment.

Methods: This single-center, prospective, cohort study included patients with hematological malignancies who underwent a first allogeneic hematopoietic stem cell transplantation between 2008 and 2014. Read More

Respir Med Case Rep 2019 4;26:244-247. Epub 2019 Feb 4.

Department of Pathology, Nagasaki University Hospital, Nagasaki, Japan.

Pleuroparenchymal fibroelastosis (PPFE) is a newly described entity of interstitial lung disease, which has been recently recognized as a rare complication of bone marrow transplantation. We report a case of 30-year-old man who developed a unique combination of pleuroparenchymal fibroelastosis with cellular and fibrotic non-specific interstitial pneumonia (NSIP) and bronchiolitis obliterans (BO) sixteen years after hematopoietic stem cell transplantation. Histological examination revealed almost exclusive infiltration of CD3-positive T lymphocytes associated with lymphoepithelial lesions and multi-focal denudation of covering epithelial cells in all components. Read More

Case Rep Oncol 2019 Jan-Apr;12(1):22-28. Epub 2019 Jan 4.

Klinik für Innere Medizin II, Hämatologie und internistische Onkologie, Universitätsklinikum Jena, Jena, Germany.

Infectious complications such as invasive aspergillosis or infection with (SM) in immunocompromised patients are associated with a high mortality rate. Our report concerns a 40-year-old male newly diagnosed very severe aplastic anemia (vSAA) who in consequence of a mosquito bite was suffering from skin lesion and consecutive soft tissue phlegmon subsequent to the administration of antithymocyte globulin; a full-thickness autologous meshed skin graft successfully performed to cover skin ulcera after allogeneic stem cell transplantation (SCT). This unusual case illustrates the importance of appropriate diagnosis, anti-infective therapy and close interdisciplinary diagnostic algorithms to minimalize side effects and the selection of resistant strains and to improve patients' outcome. Read More

Haematologica 2019 Feb 21. Epub 2019 Feb 21.

Bambino Gesù Children Hospital and Sapienza, University of Rome, Roma, Italy.

Pathophysiology of graft failure occurring after allogeneic hematopoietic stem cell transplantation still remains elusive. We measured serum levels of several different cytokines/chemokines in 15 children experiencing graft failure, comparing their values with those of 15 controls who had sustained donor cell engraftment. Already at day +3 after transplantation, patients developing graft failure had serum levels of IFNγ and CXCL9, a chemokine specifically induced by IFNγ, significantly higher than those of controls (8859±7502 versus 0 pg/ml, p=0. Read More

Haematologica 2019 Feb 21. Epub 2019 Feb 21.

UOS Milan Unit, IRGB-CNR, Milan, Italy; Humanitas Clinical and Research Center IRCCS, Rozzano, Italy;

The balance of self-renewal and differentiation is crucial to ensure the homeostasis of the hematopoietic system, and is a hallmark of hematopoietic stem cells; however, the underlying molecular pathways are not completely understood, including the role of micro-RNAs. To assess micro-RNA contributions, we performed micro-RNA profiling of hematopoietic stem cells and their immediate downstream progeny multi-potent progenitors from wild-type control and Pbx1-conditional knockout mice, whose stem cells display a profound self-renewal defect. Unsupervised hierarchical cluster analysis separated stem cells from multi-potent progenitors, suggesting that micro-RNAs might regulate the first transition step in the adult hematopoietic development. Read More

Haematologica 2019 Feb 21. Epub 2019 Feb 21.

University Hospital of Cologne, Department I of Internal Medicine, Cologne, Germany.

Blood Adv 2019 Feb;3(4):588-595

Department of Pathology and Tumor Biology, Kyoto University, Kyoto, Japan.

Subcutaneous panniculitis-like T-cell lymphoma (SPTCL) is a rare subtype of peripheral T-cell lymphoma affecting younger patients and associated with hemophagocytic lymphohistiocytosis. To clarify the molecular pathogenesis of SPTCL, we analyzed paired tumor and germline DNAs from 13 patients by whole-exome sequencing. All cases were Asians and were phenotypically sporadic with no family history of SPTCL. Read More

J Immunother Cancer 2019 Feb 21;7(1):51. Epub 2019 Feb 21.

Department of Hematology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, Hubei, China.

Background: The aggressive form of Mantle cell non-hodgkin B cell lymphoma (MCL) has a dismal prognosis. Dual targeting BTK and BCL2 with ibrutinib and venetoclax has improved outcomes in MCL patients who were predicted not to respond to conventional therapy, but it is unlikely to be curative. Chimeric antigen receptor-modified T (CAR T) cells exhibit very effective function in elimination of relapsed/refractory B-cell lymphoid malignancies, we investigated their use in a patient with relapsed MCL. Read More

Hum Immunol 2019 Feb 18. Epub 2019 Feb 18.

Indiana University School of Medicine, Department of Medicine, Division of Hematology and Oncology, Bone Marrow and Stem Cell Transplantation Program, Indiana University, Indianapolis, Indiana.

Introduction: Chromosomal abnormalities are frequent events in hematological malignancies. The degree of HLA compatibility between donor and recipient in hematopoietic stem cell transplantation is critical.

Purpose Of The Study: In this report, we describe an acute myeloid leukemia case with loss of heterozygosity (LOH) encompassing the entire HLA. Read More

Cytometry B Clin Cytom 2019 Feb 20. Epub 2019 Feb 20.

Biotherapeutics group, National Institute for Biological Standards and Control (NIBSC), Blanche Lane, South Mimms, Potters Bar, Hertfordshire, EN6 3QG, UK.

Background: Over 2,000 people a year in the United Kingdom need a bone marrow or blood stem cell transplant. It is important to accurately quantify the hematopoietic stem cells to predict whether the transplant will be successful in replenishing the immune system. However, they are present at low frequency, which complicates accurate quantification. Read More

Health Phys 2019 Apr;116(4):546-557

Indiana University, School of Medicine, Indianapolis, IN.

Accurate analyses of the delayed effects of acute radiation exposure in survivors of the hematopoietic acute radiation syndrome are hampered by low numbers of mice for examination due to high lethality from the acute syndrome, increased morbidity and mortality in survivors, high cost of husbandry for long-term studies, biological variability, and inconsistencies of models from different laboratories complicating meta-analyses. To address this, a compilation of 38 similar hematopoietic acute radiation syndrome studies conducted over a 7-y period in the authors' laboratory, comprising more than 1,500 irradiated young adult C57BL/6 mice and almost 600 day-30 survivors, was assessed for hematopoietic delayed effects of acute radiation exposure at various times up to 30 mo of age. Significant loss of long-term repopulating potential of phenotypically defined primitive hematopoietic stem cells was documented in hematopoietic acute radiation syndrome survivors, as well as significant decreases in all hematopoietic lineages in peripheral blood, prominent myeloid skew, significantly decreased bone marrow cellularity, and numbers of lineage-negative Sca-1+ cKit+ CD150+ cells (KSL CD150+; the phenotype known to be enriched for hematopoietic stem cells), and increased cycling of KSL CD150+ cells. Read More

Methods Mol Biol 2019 ;1940:129-142

Biomedical Manufacturing, CSIRO Clayton, Clayton, VIC, Australia.

The tracking of the hematopoietic potential of genetically manipulated fluorescent hematopoietic stem cells (HSC) in the bone marrow (BM) allows the assessment of regulatory processes involved in the re-establishment of hematopoiesis posttransplant. Herein, we describe the means to assess the consequence of expressing specific genes in HSC on their engraftment potential posttransplant. Read More

Front Oncol 2019 6;9:44. Epub 2019 Feb 6.

Department of Cancer Genetics with Cytogenetic Laboratory, Medical University of Lublin, Lublin, Poland.

The insertion (I allele) deletion (D allele) polymorphism of gene (rs4646994) may influence the etiopathogenesis of multiple myeloma (MM). gene is expressed in bone marrow cells and encodes angiotensin converting enzyme (ACE). It converts angiotensin I to active peptide angiotensin II, which stimulates proliferation of hematopoietic stem cells. Read More

J Korean Med Sci 2019 Feb 29;34(6):e46. Epub 2019 Jan 29.

Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.

Background: The impact of early peripheral blood chimerism on the outcome of allogeneic hematopoietic stem cell transplantation (allo-HSCT) is unclear. We aimed to determine whether day 14 peripheral blood chimerism after allo-HSCT predicts outcomes in patients with non-malignant diseases.

Methods: Data from 56 patients who received allo-HSCT between April 2007 and March 2016 were retrospectively analyzed. Read More

J Immunol 2019 Feb 20. Epub 2019 Feb 20.

Etablissement Français du Sang, 44011 Nantes Cedex 01, France.

Little is known regarding the effect of KIR/HLA incompatibilities (inc.) in the setting of T-replete haploidentical allogeneic hematopoietic stem cell transplantation using posttransplant cyclophosphamide (PTCy). In this retrospective study, the impact of KIR/HLA inc. Read More

Immunotherapy 2019 Apr;11(5):373-378

Department of Hematology, Taussig Cancer Center, Medical Oncology, Cleveland Clinic, Cleveland, OH 44195, USA.

Blinatumomab and donor lymphocyte infusion (DLI) combination is a promising cancer therapy, whereby blinatumomab might achieve an initial reduction in leukemic-cell burden using T cells, and after tumor clearance, DLI can potentially stimulate the donor immune system to achieve longer lasting remission. Here, we present a 51-year-old female with mixed phenotype acute leukemia who had a hematologic relapse 3 months after she received total body irradiation-based myeloablative allogeneic hematopoietic stem cell transplantation from an unrelated human leukocyte antigen matched (10/10) donor and achieved complete remission with minimal residual disease negativity by multi-parameter flow cytometry using the combination of blinatumomab and DLI. To the best of our knowledge, this is the first report to describe the use of blinatumomab and DLI combination therapy in the treatment of B/myeloid mixed phenotype acute leukemia. Read More

Pediatr Transplant 2019 Feb 20:e13378. Epub 2019 Feb 20.

Department of Pediatrics, Faculty of Medicine, University of Yamanashi, Yamanashi, Japan.

JMML is an aggressive hematopoietic malignancy of early childhood, and allogeneic HSCT is the only curative treatment for this disease. Umbilical cord blood is one of donor sources for HSCT in JMML patients who do not have an HLA-compatible relative, but engraftment failure remains a major problem. Here, we report two cases of JMML who were successfully rescued by HSCT from an HLA-mismatched parent after development of primary engraftment failure following unrelated CBT. Read More

Pediatr Transplant 2019 Feb 20:e13376. Epub 2019 Feb 20.

Department of Psychopathology, Avicenne Hospital, Paris XIII University & Assistance Publique-Hôpitaux de Paris, Bobigny, France.

Genoidentical HSCT is currently the only curative treatment for SCA, preventing further vascular complications in high-risk children. Studies on the psychological implications of HSCT for recipient, sibling donor, and the rest of the family have been limited in SCA. This study enrolled ten families and used semi-structured interviews to explore the parents' experience at three time points: first before transplantation, then 3 months later, and 1 year later. Read More

Br J Haematol 2019 Feb 19. Epub 2019 Feb 19.

Human Health Sciences, Graduate School of Medicine, Kyoto University, Kyoto, Japan.

The prognosis of paediatric acute myeloid leukaemia (AML) with primary induction failure (PIF) is extremely poor, and effective treatment strategies have not been established. We investigated the clinical and biological features of paediatric AML patients with PIF registered to the Japanese Paediatric Leukaemia/Lymphoma Study Group AML-05 study. The 3-year overall survival rate of the 41 PIF patients was 19. Read More

Ann Hematol 2019 Feb 19. Epub 2019 Feb 19.

Hematology Unit, Department of Pathology, Faculty of Medicine, Universiti Kebangsaan Malaysia Medical Centre, Jalan Yaacob Latif, Bandar Tun Razak, Cheras, 56000, Kuala Lumpur, Malaysia.

Short tandem repeat (STR) analysis is used in chimerism monitoring after allogeneic hematopoietic stem cell transplantation (HSCT) for patients with various hematologic malignancies. Commercial forensic STR kits often contain loci with huge differences in power of discrimination (PD) across populations, causing some loci to be less informative for chimerism analysis in certain populations. This study aimed to construct a new STR multiplex panel with highly informative loci for efficient chimerism analysis. Read More

Contemp Oncol (Pozn) 2018 31;22(4):266-269. Epub 2018 Dec 31.

Department of Pediatrics, Hematology and Oncology, Medical University of Gdansk, Gdansk, Poland.

Sinusoidal obstruction syndrome (SOS), also termed veno-occlusive disease (VOD) of the liver, is a well-known complication of haematopoietic stem cell transplantation (HSCT) both in children and adults. In the medical literature there are occasional reports of SOS in patients receiving conventional chemotherapy. In children with solid tumours this entity occurs during treatment of nephroblastoma, rhabdomyosarcoma, and medulloblastoma. Read More

Bone Marrow Transplant 2019 Feb 19. Epub 2019 Feb 19.

Department of Pediatric Hematology/Oncology/Bone Marrow Transplant, University of Colorado, Children's Hospital Colorado, Aurora, CO, USA.

High-dose chemotherapy with autologous hematopoietic stem cell transplantation (autoHSCT) is a well-established treatment for pediatric central nervous system (CNS) tumors. Given the risks of toxicity and infection, pediatric autoHSCT has been historically performed on hospitalized children. As our practice evolved, some patients were transplanted as outpatients. Read More

Bone Marrow Transplant 2019 Feb 19. Epub 2019 Feb 19.

Division of Hematology, Department of Internal Medicine, Aichi Medical University, Nagakute, Japan.

Clinical trials evaluating the role of autologous hematopoietic stem cell transplantation (auto-HCT) in multiple myeloma have mostly included patients aged <65 years. Therefore, this study was aimed to evaluate the efficacy and safety of auto-HCT in elderly patients with multiple myeloma in the era of novel agents. We retrospectively analyzed 2056 patients with multiple myeloma, who underwent auto-HCT in 2007-2014 (287 were aged ≥65 years). Read More

Euro Surveill 2019 Feb;24(7)

Instituto de Investigaciones Biomédicas August Pi i Sunyer (IDIBAPS), Facultad de Medicina, Universidad de Barcelona, Barcelona, España.

Introduction: Although human papillomavirus (HPV) routine vaccination programmes have been implemented around the world and recommendations have been expanded to include other high-risk individuals, current recommendations often differ between countries in Europe, as well as worldwide.

Aim: To find and summarise the best available evidence of HPV vaccination in high-risk patients aiding clinicians and public health workers in the day-to-day vaccine decisions relating to HPV in Spain.

Methods: We conducted a systematic review of the immunogenicity, safety and efficacy/effectiveness of HPV vaccination in high-risk populations between January 2006 and June 2016. Read More

Cells 2019 Feb 18;8(2). Epub 2019 Feb 18.

Department of Immunohematology and Blood Transfusion, L3-Q Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.

Expansion of hematopoietic stem cells (HSCs) for therapeutic purposes has been a "holy grail" in the field for many years. Ex vivo expansion of HSCs can help to overcome material shortage for transplantation purposes and genetic modification protocols. In this review, we summarize improved understanding in blood development, the effect of niche and conservative signaling pathways on HSCs in mice and humans, and also advances in ex vivo culturing protocols of human HSCs with cytokines or small molecule compounds. Read More

Medicines (Basel) 2019 Feb 18;6(1). Epub 2019 Feb 18.

Samklang Konsult, Garvargatan 17, SE 112 21 Stockholm, Sweden.

Hematopoietic stem cell transplantation (HSCT) is an established treatment for severe disorders of the pediatric hematopoietic system. However, there is a need for supportive interventions due to physiological and psychological strain. Music therapy is used in health care to help patients through difficult experiences and enable well-being. Read More

J Clin Immunol 2019 Feb 18. Epub 2019 Feb 18.

Department of Pediatric Hematology, Oncology, Blood and Marrow Transplantation, Apollo Hospitals, 320, Padma complex, Anna salai, Teynampet, Chennai, Tamil Nadu, 600035, India.

Haploidentical stem cell transplantation (haplo SCT) has emerged as an acceptable alternative to matched family donor transplantation for children diagnosed to have primary immune deficiency disorders (PIDs). We present data over 4 years on the challenges and efficacy of unmanipulated T cell replete haplo SCTs with post-transplant cyclophosphamide (PTCy) in children diagnosed to have PIDs. We performed a retrospective study in the pediatric blood and marrow transplantation unit where all children less than 18 years of age diagnosed to have PIDs and who underwent haplo SCT with PTCy from January 2014 to February 2018 were included in the study. Read More

Front Immunol 2019 4;10:23. Epub 2019 Feb 4.

Department of Pediatric Immunology and Leukocyte Biology, National Institute of Immunohaematology (ICMR), Mumbai, India.

Severe combined immunodeficiency (SCID) represents one of the most severe forms of primary immunodeficiency (PID) disorders characterized by impaired cellular and humoral immune responses. Here, we report the clinical, immunological, and molecular findings in 57 patients diagnosed with SCID from India. Majority of our patients (89%) presented within 6 months of age. Read More

Int J Biochem Cell Biol 2019 Feb 15. Epub 2019 Feb 15.

Laboratory for Cancer Biology, Cancer Institute (W.I.A), 38, Sardar Patel Road, Chennai, 600 036, Tamilnadu, India; Department of Medical Oncology and Clinical Research, Cancer Institute (W.I.A), 38, Sardar Patel Road, Chennai, 600 036, Tamilnadu, India. Electronic address:

Stem cells or Cancer stem cells (CSCs) have now been identified in different type of tissues by using surface markers. Functional assays such as ALDEFLUOR and side population which are marker independent have been additional approaches. However, whether all these approaches identify the same population of cells remain uncertain. Read More

Indian Dermatol Online J 2019 Jan-Feb;10(1):50-53

Department of General Medicine, St. John's Medical College, Bengaluru, Karnataka, India.

Scleromyxedema is a rare progressive cutaneous mucinosis of unknown etiology with equal prevalence in both men and women. It is usually associated with monoclonal gammopathy in most of the cases. Various treatment modalities have been tried for scleromyxedema including steroids, intravenous immunoglobulin (IVIg), autologous hematopoietic stem cell transplantation, and melphalan, but none has proved to be fully effective. Read More

Int J Hematol Oncol Stem Cell Res 2018 Oct;12(4):322-328

Diagnostic Laboratory Sciences and Technology Research Center, School of Paramedical Sciences, Shiraz University of Medical Sciences, Shiraz, Iran.

Hematopoietic stem cell transplantation (HSCT) is a useful treatment. In contrast to solid organ transplantations, the use of ABO blood group mismatch is acceptable in HSCT. Immediate or late hemolytic reactions, pure red cell aplasia, delayed red blood cell recovery, and graft-versus -host disease are the results of this situation. Read More

Int J Hematol Oncol Stem Cell Res 2018 Oct;12(4):282-290

Jinnah Sindh Medical University, Karachi, Pakistan.

Cardiac muscle possesses a limited capacity to regenerate its tissue on its own. It is less likely to reverse the altered cardiac functioning to its normal physiological state after a major myocardial infarction. Stem cell transplantation provided a unique therapeutic approach in managing such injuries. Read More

Oxid Med Cell Longev 2019 10;2019:8727925. Epub 2019 Jan 10.

Department of Clinical Genetics, Medical University of Lublin, Lublin, Poland.

The gene encodes a survivin protein belonging to class III of inhibitors of apoptosis, IAP. This protein serves a dual role. First, it regulates cell death, and second, it is an important regulator of mitosis progression, although its physiological regulatory function has not been fully understood. Read More

Front Immunol 2019 1;10:93. Epub 2019 Feb 1.

Fels Institute for Cancer Research and Molecular Biology, Temple University, Philadelphia, PA, United States.

Graft-vs.-host disease (GVHD) remains a significant cause of morbidity and mortality after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Significant progresses have been made in defining the dichotomous role of dendritic cells (DCs) in the development of GVHD. Read More

Am J Hematol 2019 Feb 17. Epub 2019 Feb 17.

Hematology, Oncology & Cardiovascular Medicine, Kyushu University Hospital, Fukuoka, Japan.

Quintessence Int 2019 ;50(3):208-213

Acute myeloid leukemia is a bone marrow malignancy in which blasts count increases by more than 20% in the bone marrow. Allogeneic hematopoietic stem cell transplantation (alloHCT) is a treatment option for these patients with high risk of graft versus host disease (GVHD) development. Chronic GVHD (cGVHD) often mimics a variety of autoimmune conditions such as systemic lupus erythematous or systemic sclerosis. Read More

Leuk Lymphoma 2019 Feb 18:1-9. Epub 2019 Feb 18.

a Peking University People's Hospital , Peking University Institute of Hematology , Beijing , China.

Acute myeloid leukemia (AML) patients with biallelic CEBPA (bi CEBPA) mutations are considered prognostically favorable, but 38-58% of them still relapse. Therefore, recognizing patients with a high risk of relapse is important. We retrospectively analyzed 83 bi CEBPA AML. Read More

Clin Infect Dis 2019 Feb 17. Epub 2019 Feb 17.

BMJ Open 2019 Feb 15;9(2):e026093. Epub 2019 Feb 15.

Département des Maladies Infectieuses et Tropicales, Hospices Civils de Lyon, Lyon, France.

Introduction: Immune reconstitution after haematopoietic stem cell transplantation (HSCT) is a complex and dynamic process, varying from a state of nearly complete immunosuppression to an expected full immune recovery. Specific vaccination guidelines recommend reimmunisation after HSCT but data regarding vaccine efficacy in this unique population are scarce. New immune functional assays could enable prediction of vaccine response in the setting of HSCT. Read More

Biol Blood Marrow Transplant 2019 Feb 14. Epub 2019 Feb 14.

Nemours/ Alfred I. duPont Hospital for Children, Wilmington, DE, USA; Department of Pediatrics, Shimane University Faculty of Medicine, Shimane, Japan; Department of Pediatrics, Graduate School of Medicine, Gifu University, Gifu, Japan; Department of Pediatrics, Thomas Jefferson University, Philadelphia, PA. Electronic address:

Allogenic hematopoietic stem cell transplantation (HSCT) has been shown to be a treatment option for a selected group of patients with mucopolysaccharidoses (MPS) (MPS I, II, IVA, VI, and VII). Early diagnosis and timely referral to an expert in MPS are critical, followed by a complete examination and evaluation with a multidisciplinary team, including a transplant physician. Treatment recommendations for MPS are based on multiple factors such as biological, sociological, and financial effects. Read More

Biol Blood Marrow Transplant 2019 Feb 14. Epub 2019 Feb 14.

Section of Oncology/BMT, Departments of Oncology and Pediatrics, Alberta Children's Hospital, University of Calgary, Calgary, Canada.

Sickle cell disease (SCD) is a potentially debilitating hemoglobinopathy associated with early mortality. The only established curative therapy is hematopoietic cell transplantation (HCT)with a matched sibling donor (MSD). The National Institutes of Health (NIH) nonmyeloablative regimen of alemtuzumab/300 cGy total body irradiation and prolonged sirolimus exposure for graft-versus-host disease (GVHD) prophylaxis was administered to 16 children and adolescents. Read More

Bone Marrow Transplant 2019 Feb 15. Epub 2019 Feb 15.

Institut de Cancérologie Lucien Neuwirth, Saint-Etienne, France.

Several approaches have been developed to overcome historical barriers associated with poor outcomes in the setting of HLA-haploidentical allogeneic transplantation (HaploSCT). Here, we examine the outcome of patients with various hematological disorders undergoing HaploSCT with high-dose, post-transplantation cyclophosphamide. We performed a retrospective study on 381 patients from 30 centers between January 2013 and December 2015. Read More

Molecules 2019 Feb 15;24(4). Epub 2019 Feb 15.

National Institutes for Food and Drug Control, No. 2, Tiantan Xili, Dongcheng District, Beijing 100050, China.

The therapeutic recombinant human keratinocyte growth factor 1 (rhKGF-1) was approved by the FDA for oral mucositis resulting from hematopoietic stem cell transplantation for hematological malignancies in 2004. However, no recommended bioassay for rhKGF-1 bioactivity has been recorded in the U.S. Read More

Biol Blood Marrow Transplant 2019 Feb 12. Epub 2019 Feb 12.

Division of Hematology/Oncology, Hospital for Sick Children, Toronto, Ontario.

Introduction: Access to hematopoietic stem cell transplantation (HSCT) in pediatric acute lymphoblastic leukemia (ALL) is primarily dependent on disease-related factors but may be influenced by social and economic determinants.

Methods: We included all children < 15 years old with newly diagnosed ALL in Canada between 2001 and 2018 using the Cancer in Young People in Canada (CYP-C) national registry. We examined factors potentially associated with the likelihood of receiving HSCT using univariate and multivariable logistic regression models. Read More

Cancer Sci 2019 Feb 15. Epub 2019 Feb 15.

Medical Innovation Center, Graduate School of Medicine, Kyoto University, Sakyo-ku, Kyoto, 606-8507, Japan.

Adult long-term hematopoiesis depends on sustaining hematopoietic stem/progenitor cells (HSPCs) in bone marrow (BM) niches, where their balance of quiescence, self-renewal, and hematopoietic differentiation is tightly regulated. While various BM stroma cells that produce niche factors have been identified, regulation of the intrinsic responsiveness of HSPCs to the niche factors remains elusive. We previously reported that mice deficient for Sipa1, a Rap1 GTPase-activating protein, develop diverse hematopoietic disorders of late onset. Read More