Search Our Scientific Publications & Authors

J Allergy Clin Immunol Pract 2019 Apr 14. Epub 2019 Apr 14.

Non-communicable Diseases Research Center, Alborz University of Medical Sciences, Karaj, Iran. Electronic address:

Background: LPS-responsive beige-like anchor protein (LRBA) deficiency is a primary immunodeficiency and immune dysregulation syndrome caused by biallelic mutations in the LRBA gene. These mutations usually abrogate the protein expression of LRBA, leading to a broad spectrum of clinical phenotypes including autoimmunity, chronic diarrhea, hypogammaglobulinemia and recurrent infections.

Objective: Our aim was to systematically collect all studies reporting on the clinical manifestations, molecular and laboratory findings, and management of patients with LRBA deficiency. Read More

Cell Rep 2019 Apr;27(3):719-729.e6

Cancer Research UK Cambridge Institute, Li Ka Shing Centre, University of Cambridge, Cambridge CB2 0RE, UK; Watson School of Biological Sciences, Howard Hughes Medical Institute, Cold Spring Harbor Laboratory, Cold Spring Harbor, NY 11724, USA; New York Genome Center, New York, NY 10013, USA. Electronic address:

Long non-coding RNAs (lncRNAs) show patterns of tissue- and cell type-specific expression that are very similar to those of protein coding genes and consequently have the potential to control stem and progenitor cell fate decisions along a differentiation trajectory. To understand the roles that lncRNAs may play in hematopoiesis, we selected a subset of mouse lncRNAs with potentially relevant expression patterns and refined our candidate list using evidence of conserved expression in human blood lineages. For each candidate, we assessed its possible role in hematopoietic differentiation in vivo using competitive transplantation. Read More

N Engl J Med 2019 Apr;380(16):1525-1534

From the Departments of Bone Marrow Transplantation and Cellular Therapy (E.M., B.T., W.J., S.G.), Hematology (S.Z., Z.M., J.C., J.D., X.T., B.Y.R., M.J.W., B.P.S.), Therapeutics Production and Quality (T.L., M.M.M.), Immunology (H.A., B.Y.), Pharmaceutical Sciences (S.J.C.), Biostatistics (G.K., C.L.), and Infectious Diseases (G.M.), St. Jude Children's Research Hospital, Memphis, TN; the Allergy and Clinical Immunology Division, Hospital Nacional Edgardo Rebagliati Martins, Lima, Peru (J.C.A.B.); the Department of Pediatrics, Allergy-Immunology Division, Children's Hospital Los Angeles, Los Angeles (J.A.C.), and the Department of Pediatrics, Division of Pediatric Allergy-Immunology-Bone Marrow Transplantation, University of California, San Francisco (UCSF) Benioff Children's Hospital, San Francisco (J.R.L.-B., J.M.P., M.J.C.) - both in California; the Department of Pediatrics, Pediatric Allergy and Immunology, University of New Mexico, Albuquerque (E.D.); University of Oklahoma Health Sciences Center, Tulsa (J.T.L.); Departamento de Pediatria da Universidade de Taubaté, Conselho Nacional de Medicina, São Paulo (A.C.M.A.); Copperfield Childcare, Claremont, South Africa (H.W.); and the Genetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD (S.S.D.R., H.L.M.).

Background: Allogeneic hematopoietic stem-cell transplantation for X-linked severe combined immunodeficiency (SCID-X1) often fails to reconstitute immunity associated with T cells, B cells, and natural killer (NK) cells when matched sibling donors are unavailable unless high-dose chemotherapy is given. In previous studies, autologous gene therapy with γ-retroviral vectors failed to reconstitute B-cell and NK-cell immunity and was complicated by vector-related leukemia.

Methods: We performed a dual-center, phase 1-2 safety and efficacy study of a lentiviral vector to transfer complementary DNA to bone marrow stem cells after low-exposure, targeted busulfan conditioning in eight infants with newly diagnosed SCID-X1. Read More

J Pediatr Hematol Oncol 2019 Apr 15. Epub 2019 Apr 15.

Department of Pediatrics, National Center for Global Health and Medicine.

Chronic myeloid leukemia (CML) is commonly associated with major BCR-ABL transcript. We present a child with blastic phase CML associated with minor BCR-ABL transcript without prior CML diagnosis. Diagnosis was achieved by fluorescence in situ hybridization of peripheral blood neutrophils, which identified 90% as BCR-ABL positive. Read More

Transpl Infect Dis 2019 Apr 16:e13095. Epub 2019 Apr 16.

Pediatric Clinic, "P & A Kyriakou" Children's Hospital, National and Kapodistrian University of Athens, Faculty of Nursing, Athens, Greece.

Background: Viral infections are a significant cause of morbidity and mortality in pediatric transplant populations. We analyzed the epidemiology of viral infections in pediatric hematopoietic stem cell transplant (HSCT) patients, including their incidence, associated risk factors, and outcome.

Methods: In a prospective study from September 2011 to September 2015 blood, urine and stool specimens were monitored weekly from transplantation to day 100 or after if clinically suspected, by use of real-time polymerase chain reaction. Read More

Eur J Drug Metab Pharmacokinet 2019 Apr 16. Epub 2019 Apr 16.

Department of Physical Pharmacy and Pharmacokinetics, Poznan University of Medical Sciences, 6 Święcickiego Street, 60-781, Poznań, Poland.

Background And Objectives: Treosulfan (dihydroxybusulfan), licensed for the treatment of ovarian carcinoma, is investigated in clinical trials as a myeloablative agent for conditioning prior to hematopoietic stem cell transplantation. Clinical experience shows that treosulfan exhibits lower organ toxicity than busulfan, including hepatotoxicity. Elimination of busulfan primarily via enzymatic conjugation with glutathione (GSH) in the liver is considered to be the main cause of the drug's hepatotoxicity and interpatient clearance variability. Read More

Ann Hematol 2019 Apr 16. Epub 2019 Apr 16.

Collegium Medicum UMK, University Hospital, Bydgoszcz, Poland.

It has been shown recently that donor and/or recipient cytomegalovirus (CMV) seropositivity is associated with a significant overall survival (OS) decline in acute leukemia patients who underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT). We now analyzed the prognostic impact of the donor/recipient CMV serostatus in 6968 patients with chronic hematological malignancies who underwent allo-HSCT. Donor and/or recipient CMV seropositivity was associated with a significantly reduced 2-year progression-free survival (PFS, 50% vs. Read More

Ann Hematol 2019 Apr 16. Epub 2019 Apr 16.

Department of Hematology, Changhai Hospital, The Second Military Medical University, Shanghai, China.

To investigate the effect of chronic graft-versus-host disease (cGVHD) on the outcomes of acute myeloid leukemia (AML) patients who relapsed after allogenic hematopoietic cell transplantation, we performed a retrospective analysis on 218 patients with a median follow-up of 21.4 (3.4-179. Read More

Ann Hematol 2019 Apr 16. Epub 2019 Apr 16.

Department of Plastic, Reconstructive and Hand Surgery, University Hospital, Regensburg, Germany.

Refractory skin ulcers due to severe chronic graft-versus-host disease (cGVHD) remain to be associated with significant morbidity and mortality.We performed an allogeneic donor skin transplantation in seven adult patients after allogeneic hematopoietic stem cell transplantation for cGVHD-associated refractory skin ulcers. While four patients received a split skin graft (SSG), in one patient, a full thickness skin graft for two small refractory ulcers of the ankle was performed, and one patient received in vitro expanded donor keratinocyte grafts derived from hair roots of the original unrelated donor. Read More

Blood 2019 Apr 16. Epub 2019 Apr 16.

Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY, United States

Cancer is the leading cause of death for HIV-infected persons in economically developed countries, even in the era of antiretroviral therapy (ART). Lymphomas remain a leading cause of cancer morbidity and mortality for HIV-infected patients and have increased incidence even in patients optimally treated with ART. Even limited interruptions of ART can lead to cluster of differentiation 4 (CD4) cell nadirs and HIV viremia, and increase the risk of lymphoma. Read More

Int J Hematol 2019 Apr 13. Epub 2019 Apr 13.

Hematology Division, Tokyo Metropolitan Cancer and Infectious Diseases Center, Komagome Hospital, 3-18-22 Honkomagome, Bunkyo-ku, Tokyo, 113-8677, Japan.

Autologous stem-cell transplantation is an effective procedure for the treatment of multiple myeloma, and involves the collection of hematopoietic stem cells (HSCs). However, in some patients, HSCs in the bone marrow fail to mobilize. Pomalidomide upregulates CXCR4 in hematopoietic stem cells, in a manner similar to that of lenalidomide, and is, thus, likely to have a negative impact on hematopoietic stem-cell mobilization in multiple myeloma patients. Read More

Tissue Eng Regen Med 2019 Apr 21;16(2):107-118. Epub 2019 Feb 21.

3Department of Neurosurgery, The China-Japan Union Hospital of Jilin University, No. 126 Xiantai Street, Changchun, 130033 China.

Background: Liver disease is one of the top causes of death globally. Although liver transplantation is a very effective treatment strategy, the shortage of available donor organs, waiting list mortality, and high costs of surgery remain huge problems. Stem cells are undifferentiated cells that can differentiate into a variety of cell types. Read More

Indian J Hematol Blood Transfus 2019 Apr 7;35(2):233-239. Epub 2019 Jan 7.

2Department of Hematology, All India Institute of Medical Sciences (AIIMS), New Delhi, 110029 India.

Acute graft-versus-host disease (aGVHD) and relapse are major issues for patients undergoing allogenic hematopoietic stem cell transplant (allo-HSCT). T-regulatory (Treg) cells in the donor graft are negatively correlated with the incidence of aGVHD without any impact on relapse. In this study to determine the association of Treg cells with aGVHD in allo-HSCT patients. Read More

Bone Marrow Transplant 2019 Apr 15. Epub 2019 Apr 15.

Department of Hematopoietic Stem Cell Transplantation, Dmitriy Rogachev National Medical Research Сenter of Pediatric Hematology, Oncology and Immunology, Samory Mashela street, 1, Moscow, 117997, Russia.

Proc Natl Acad Sci U S A 2019 Apr 15. Epub 2019 Apr 15.

State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, National Research Center for Translational Medicine, Ruijin Hospital affiliated with Shanghai Jiao Tong University School of Medicine, 200025 Shanghai, China;

Relapsed and refractory (R/R) multiple myeloma (MM) patients have very poor prognosis. Chimeric antigen receptor modified T (CAR T) cells is an emerging approach in treating hematopoietic malignancies. Here we conducted the clinical trial of a biepitope-targeting CAR T against B cell maturation antigen (BCMA) (LCAR-B38M) in 17 R/R MM cases. Read More

Diseases 2019 Apr 4;7(2). Epub 2019 Apr 4.

Department of Clinical Immunology and Rheumatology, Hannover Medical School, 30625 Hannover, Germany.

Immunodeficiency, centromeric instability and facial anomalies syndrome 2 (ICF2) is a rare autosomal recessive primary immunodeficiency disorder. So far, 27 patients have been reported. Here, we present three siblings with ICF2 due to a homozygous ZBTB24 gene mutation (c. Read More

Exp Hematol 2019 Apr 12. Epub 2019 Apr 12.

Department of Hematology, Cancer Research Center Groningen, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands. Electronic address:

Reduced expression of the transcription factor PU.1 is frequently associated with development of acute myeloid leukemia (AML), whereas elevated levels of CITED2 (CBP/p300-interacting-transactivator-with-an-ED-rich-tail 2) enhance maintenance of both normal and leukemic hematopoietic stem and progenitor cells (HSPCs). Recent findings indicated that PU. Read More

Exp Hematol 2019 Apr 12. Epub 2019 Apr 12.

National Heart, Lung and Blood Institute (NHLBI), National Institutes of Health (NH), Bethesda, MD 20892, USA. Electronic address:

A causal link between hematopoietic stem/progenitor cell (HSPC) dysfunction and DNA damage accrual has been proposed. Clinically relevant strategies to maintain genome integrity in these cells are needed. Here we report that eltrombopag, a small molecule agonist of the thrombopoietin (TPO) receptor used in the clinic, promotes DNA double strand break (DSB) repair in human HSPCs. Read More

Front Pediatr 2019 28;7:107. Epub 2019 Mar 28.

Division of Pediatric Allergy and Immunology, Department of Pediatrics, Penn State Milton S. Hershey Medical Center, Hershey, PA, United States.

Chronic Granulomatous Disease (CGD) is caused by genetic defects in the phagocyte NADPH oxidase leading to potentially severe infections with catalase positive micro-organisms. With the innate immune system being affected this disease usually presents before the age of 5 years with infections involving the skin, lung, liver or lymphnodes. Infections with specific catalase positive organisms, especially and prompt a workup for CGD in affected patients. Read More

Front Immunol 2019 29;10:621. Epub 2019 Mar 29.

Center for Cancer and Immunology Research, Children's National Health System, Washington, DC, United States.

Mycobacterial Infections can be severe in patients with T-cell deficiency or phagocyte disorders, and treatment is frequently complicated by antimicrobial resistance. Restoration of T-cell immunity via stem cell transplantation facilitates control of mycobacterial infections, but presence of active infections during transplantation is associated with a higher risk of mortality. Adoptive T cell immunotherapy has been successful in targeting viruses, but has not been attempted to treat mycobacterial infections. Read More

Cell Transplant 2019 Apr 15:963689719842162. Epub 2019 Apr 15.

2 Department of Clinical Immunology, School of Laboratory Medicine, Xinxiang Medical University, China.

Human CD133 stem cells were injected into the bone marrow cavity of NOG (NOD Shi-SCID IL2Rγc) mice with or without preconditioning of busulfan in order to assess the efficiency of human CD133 cells engraftment. Peripheral blood from CD133-engrafted NOG mice was analyzed by flow cytometry. The results showed that human CD19 B lymphocytes could be detected at 4 weeks post-transplantation, and human CD4, CD8 subsets of T lymphocytes, CD19 CD14 HLA-DR DCs and CD19 CD14 monocytes could be detected at 16 weeks post-transplantation. Read More

Int Wound J 2019 Apr 14. Epub 2019 Apr 14.

Department of Dermatology, Medical Faculty, Ege University, Izmir, Turkey.

Extranodal NK/T cell lymphoma (ENKTL) is a rare form of non-Hodgkin lymphoma. It mostly occurs in the upper respiratory tract. Cutaneous involvement can be seen among the extranasal ENKTLs. Read More

J Med Virol 2019 Apr 14. Epub 2019 Apr 14.

Laboratory of Virology and Cell Culture, Instituto de Patologia Tropical e Saúde Pública, Universidade Federal de Goiás, Goiânia, GO, Brazil.

Objectives: The aim of this study was to evaluate the occurrence of human bocavirus (HBoV) and to determine viral loads in samples of patients admitted for allogeneic hematopoietic stem cell transplantation (allo-HSCT).

Methods: Fecal and serum samples were collected from 19 patients, during a 24-month period. Samples were screened by qPCR TaqMan assay, with specific probe and primers targeting the NP1 gene of all HBoVs genotypes (HBoV-1 to -4), and viral loads were determined using serial dilutions of a recombinant plasmid. Read More

Dig Dis Sci 2019 Apr 13. Epub 2019 Apr 13.

Division of Hematology, Department of Internal Medicine, College of Medicine, The Catholic University of Korea, Seoul, Republic of Korea.

Background And Aims: Long-term results on hepatitis B virus (HBV) reactivation in patients with resolved infection during anti-cancer therapy are unknown. This study investigated long-term risk and therapeutic endpoints including hepatitis B surface antigen (HBsAg) seroclearance following antiviral therapy in patients developing reactivation of resolved HBV.

Methods: The study included 528 consecutive HBsAg-negative/hepatitis B core antibody-positive patients who underwent rituximab treatment or hematopoietic stem cell transplantation (HSCT) between 2006 and 2016. Read More

Clin Infect Dis 2019 Jan 15. Epub 2019 Jan 15.

Department of Hematology, King's College Hospital NHS Foundation Trust, Denmark Hill.

Introduction: Autologous hematopoietic stem cell transplantation (AHSCT) with anti-thymocyte globulin (ATG) conditioning as treatment of active multiple sclerosis (MS) is rapidly increasing across Europe (EBMT registry data 2017). Clinically significant Epstein-Barr virus reactivation (EBV-R) following AHSCT with ATG for severe autoimmune conditions is an underrecognized complication relative to T-cell deplete transplants performed for hematological diseases. This retrospective study reports EBV-R associated significant clinical sequelae in MS patients undergoing AHSCT with rabbit ATG. Read More

Glia 2019 Jun 11;67(6):1196-1209. Epub 2019 Feb 11.

Department of Neuropathology, University Medical Center Göttingen, Göttingen, Germany.

X-linked adrenoleukodystrophy (X-ALD) and metachromatic leukodystrophy (MLD) are two relatively common examples of hereditary demyelinating diseases caused by a dysfunction of peroxisomal or lysosomal lipid degradation. In both conditions, accumulation of nondegraded lipids leads to the destruction of cerebral white matter. Because of their high lipid content, oligodendrocytes are considered key to the pathophysiology of these leukodystrophies. Read More

Blood Cancer J 2019 Apr 12;9(4):46. Epub 2019 Apr 12.

EBMT Paris Study Office/CEREST-TC, Paris, France.

The role of umbilical cord blood transplantation (CBT) in acute myeloid leukemia (AML) patients with active disease at allogeneic hematopoietic cell transplantation (allo-HCT) remains poorly investigated. In this study, we compared transplantation outcomes of 2963 patients with primary refractory or relapsed AML given CBT, 10/10 HLA-matched UD, or 9/10 HLA-matched UD allo-HCT from 2004 to 2015 at EBMT-affiliated centers. Neutrophil engraftment and complete remission rates in CBT, UD 10/10, and UD 9/10 recipients were 75 and 48%, 93 and 69%, and 93 and 70%, respectively. Read More

Clin J Am Soc Nephrol 2019 Apr 12. Epub 2019 Apr 12.

National Clinical Research Center of Kidney Diseases, Jinling Hospital, Nanjing University School of Medicine, Nanjing, China.

Background And Objectives: Our study evaluated the efficiency and safety of autologous hematopoietic stem cell transplantation treatment for patients with refractory lupus nephritis.

Design, Setting, Participants, & Measurements: From July 2011 to January 2015, a total of 22 patients with refractory lupus nephritis were enrolled in this study. Peripheral blood stem cells were mobilized with cyclophosphamide and granulocyte colony stimulating factor and reinfused after treatment with cyclophosphamide and antithymocyte globulin. Read More

Transplant Proc 2019 Apr 26;51(3):998-1001. Epub 2019 Jan 26.

Department of Hematology, Faculty of Medicine, Hokkaido University, Sapporo, Japan.

Objective: To elucidate the mechanisms by which orally administered tacrolimus was not absorbed in a patient following allogeneic hematopoietic stem cell transplantation.

Clinical Course: A 17-year-old girl with acute myeloid leukemia underwent HLA-haploidentical peripheral blood stem cell transplantation following fludarabine, busulfan, and total-body irradiation. Graft-vs-host disease prophylaxis was post-transplant cyclophosphamide, followed by intravenous tacrolimus and mycophenolate mofetil. Read More

Transplant Proc 2019 Apr 31;51(3):896-900. Epub 2019 Jan 31.

Third Department of Internal Medicine, Yamaguchi University School of Medicine, Ube, Yamaguchi, Japan.

Allogeneic hematopoietic cell transplant (HCT) is a curative procedure for myeloid malignant neoplasms, but relapse after HCT remains critical. A conditioning regimen involving granulocyte colony-stimulating factor-combined high-dose cytarabine, cyclophosphamide, and total body irradiation (G-CSF-combined high-dose cytarabine/cyclophosphamide/total-body irradiation [HDCA/CY/TBI]) was reported to improve outcomes after cord blood transplant (CBT) for myeloid malignant neoplasms, but this regimen was not previously evaluated among patients undergoing bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT).

Methods: We retrospectively analyzed 28 patients who underwent allogeneic HCT including BMT from a related (1 patient) or unrelated donor (9 patients), PBSCT from a related donor (7 patients), or single-unit CBT from an unrelated donor (11 patients) after a G-CSF-combined HDCA/CY/TBI regimen. Read More

Transplant Proc 2019 Apr 9;51(3):890-895. Epub 2019 Jan 9.

Department of Hematology, The Second Affiliated Hospital of Chongqing Medical University, Chongqing, P.R. China. Electronic address:

Graft-vs-host disease (GVHD) is one of the biggest challenges in haploidentical hematopoietic stem cell transplantation. Antithymocyte globulins (ATGs) are widely used to overcome GVHD, but excessive immunosuppression increases the chances of relapse and infection following transplantation. No defined standard of the appropriate dose of ATG usage is recognized. Read More

Transplant Proc 2019 Apr 8;51(3):880-889. Epub 2019 Jan 8.

Department of Transplantation, Jagiellonian University Medical College, Krakow, Poland.

Application of mesenchymal stem cells (MSC) enables a novel approach to the therapy of graft- vs-host disease (GVHD) after hematopoietic stem cell transplantation. Herein we present our preliminary experience with the use of allogeneic bone marrow‒derived MSC in 9 pediatric patients after hematopoietic transplantation complicated by severe acute or chronic GVHD (aGVHD, cGVHD) resistant to steroids and second-line immunosuppressants. The MSC therapy was applied concurrently with immunosuppressive treatment in 5 patients as a single infusion, in four patients as 2-6 infusions. Read More

Leuk Res 2019 Apr 4;81:1-9. Epub 2019 Apr 4.

Division of Hematology, Department of Medicine, Mayo Clinic, 200 First St. SW, Rochester, MN 55905, United States; Department of Medical Oncology, Mayo Clinic 200 First St. SW, Rochester, MN, 55905.

The infusion of autograft absolute lymphocyte and monocyte counts affect survival in patients undergoing autologous peripheral hematopoietic stem cell transplantation (APHSCT). However, the specific autograft immune effector cells affecting survival post-APHSCT are unknown. Thus, we performed an ad hoc analysis from our published double-blind, randomized phase III clinical trial in non-Hodgkin's lymphoma (NHL) patients, looking at the infused autograft immune effector cells and their relationship with clinical outcomes post-APHSCT. Read More

Hematol Oncol Stem Cell Ther 2019 Apr 6. Epub 2019 Apr 6.

Division of Hematology-Oncology and Blood and Marrow Transplantation Program, Mayo Clinic, Jacksonville, FL, USA.

Lymphomatous involvement of the ocular adnexal region (OAR) is generally rare; however, it constitutes the most common ocular malignancy. Most cases of ocular adnexal lymphomas are extra nodal marginal zone B cell lymphomas. Mantle cell lymphoma involvement (MCL) of the OAR is a less common presentation compared with other lymphomas and it has been reported anecdotally. Read More

Hematol Oncol Stem Cell Ther 2019 Apr 6. Epub 2019 Apr 6.

Raisa Gorbacheva Memorial Research Institute of Children Oncology, Hematology and Transplantation, Department of Hematology, Transfusiology, Transplantation, Faculty of Postgraduate Education, Pavlov First Saint Petersburg State Medical University, Saint-Petersburg, Russia.

Background: Allogeneic hematopoietic stem cell transplantation (alloHSCT) is the only treatment option with curative potential in patients with myelofibrosis (MF). The aim of our study was to evaluate the safety of splenectomy before alloHSCT in MF patients who failed to achieve significant spleen response after ruxolitinib therapy.

Methods: Splenectomy was performed in 12 patients for alloHSCT with myelofibrosis-primary (6 patients), post-polycythemia vera (3 patients). Read More

Pediatr Hematol Oncol 2019 Apr 12:1-17. Epub 2019 Apr 12.

c Host Research Team EA4607 SNA-EPIS, PRES Lyon, Jean Monnet University, University Hospital , Saint-Etienne , France.

This study consists of a retrospective study including 71 childhood leukemia survivors (36 females) treated with allo-HSCT 12 Gy fractionated total body irradiation (fTBI) conditioning, with a median age of 25.0 y at time of follow-up and a median delay of 14.8 y since the graft. Read More

Stem Cells 2019 Apr 12. Epub 2019 Apr 12.

Department of Pharmaceutical Sciences, School of Pharmacy, West Virginia University, Morgantown, WV.

Emerging evidence have shown that resting quiescent hematopoietic stem cells (HSCs) prefer to utilize anaerobic glycolysis rather than mitochondrial respiration for energy production. Compelling evidence has also revealed that altered metabolic energetics in HSC underlies the onset of certain blood diseases. However, the mechanisms responsible for energetic reprogramming remain elusive. Read More

Mol Ther Methods Clin Dev 2019 Jun 2;13:321-333. Epub 2019 Mar 2.

Stem Cell and Neurotherapies, Faculty of Medical and Human Sciences, University of Manchester, Manchester, UK.

Enzyme replacement therapy with laronidase is an established treatment for Mucopolysaccharidosis type I (MPS I), but its efficacy may be limited by the development of anti-drug antibodies, which inhibit cellular uptake of the enzyme. In a related disorder, infantile Pompe disease, immune tolerance induction with low-dose, short-course methotrexate appears to reduce antibody formation. We investigated a similar regimen using oral methotrexate in three MPS I patients. Read More

BMC Cancer 2019 Apr 11;19(1):347. Epub 2019 Apr 11.

Department of Rehabilitation Medicine, Kyungpook National University Hospital, 130 Dongdeok-ro, Jung-gu, Daegu, 41944, South Korea.

Background: Hematopoietic stem cell transplantation (HSCT) remains an important therapeutic option for many hematologic malignancies. Bone marrow harvesting from an appropriate donor must be conducted for hematopoietic stem cell transplantation (HSCT). Many previous studies show complications of the recipient after hematopoietic stem cell transplantation (HSCT). Read More

Pediatr Transplant 2019 Apr 11:e13416. Epub 2019 Apr 11.

The Child and Adolescent Clinic, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark.

Graft-versus-host disease (GVHD) is a main cause of morbidity and mortality following hematopoietic stem cell transplantation. The cumulative incidence of acute and chronic GVHD (aGVHD, cGVHD) reaches 30%-50% and 20% in pediatric populations, respectively. Prednisolone and/or calcineurin inhibitors (CNI) are first-line treatments, but no superior second-line treatment has yet been established. Read More

J Pediatr Hematol Oncol 2019 Apr 9. Epub 2019 Apr 9.

Division of Pediatric Hematology-Oncology, Medical College of Wisconsin, Milwaukee, WI.

CD19 chimeric antigen receptor T-cell (CART) therapy has revolutionized the treatment of patients with relapsed/refractory hematologic malignancies, especially B-cell acute lymphoblastic leukemia. As CART immunotherapy expands from clinical trials to FDA-approved treatments, a consensus among oncologists and hematopoietic cell transplant (HCT) physicians is needed to identify which patients may benefit from consolidative HCT post-CART therapy. Here, we review CD19 CART therapy and the outcomes of published clinical trials, highlighting the use of post-CART HCT and the pattern of relapse after CD19 CART. Read More

Transpl Infect Dis 2019 Apr 10:e13089. Epub 2019 Apr 10.

Department of Pediatric Hematology and Oncology, University Children's Hospital Muenster.

Cytomegalovirus retinitis (CMVR) may occur after allogeneic hematopoietic stem cell transplantation (HSCT). However, little is known about its incidence, strategies for ophthalmic surveillance, and timely implementation of adequate antiviral treatment in pediatric allogeneic HSCT recipients. We provide a retrospective analysis of the epidemiology and clinical features of CMVR in pediatric allogeneic HSCT patients transplanted at our center over a 16-year period. Read More

Transpl Infect Dis 2019 Apr 10:e13091. Epub 2019 Apr 10.

Division of Infectious Diseases, University of North Carolina, Chapel Hill, NC, USA.

Current guidelines recommend that hepatitis C virus (HCV) infection be treated completely prior to hematopoietic cell transplantation or delayed until immune reconstitution after transplantation to avoid drug-drug interactions and treatment interruption. However, these recommendations were informed by outcomes using treatment with ribavirin and pegylated interferon. We report the first case of successful treatment of HCV using direct-acting antivirals during hematopoietic cell transplantation. Read More

Artif Organs 2019 Apr 10. Epub 2019 Apr 10.

Henan Institute of Haematology, Department of Hematology, Henan Cancer Hospital, The Affliated Cancer Hospital of Zhengzhou University, Zhengzhou, China.

Objective: This study aims to review the clinical efficacy and factors affecting the treatment of multiple myeloma (MM) by autologous hematopoietic stem cell transplantation (ASCT).

Methods: The clinical data of 47 patients with MM from the Department of Hematology of Henan Cancer Hospital from September 2010 to July 2018 were retrospectively analyzed.

Results: At pre-transplantation of autologous cells, 25. Read More

Int J Hematol 2019 Apr 10. Epub 2019 Apr 10.

Department of Hematology and Oncology, Konan Kosei Hospital, 137 Omatsubara, Takaya-cho, Konan, Aichi, 483-8704, Japan.

Cryptogenic organizing pneumonia (COP) after allogeneic hematopoietic stem cell transplantation (HSCT) is characterized by frequent recurrence. Few studies have examined onset and recurrence patterns of COP after HSCT. We investigated the clinical features of COP after HSCT in a single-center retrospective study including 165 consecutive patients who underwent allogeneic HSCT. Read More

Am J Transl Res 2019 15;11(3):1299-1310. Epub 2019 Mar 15.

Department of Hematology, The Third Affiliated Hospital of Chongqing Medical University (Gener Hospital) Chongqing 401120, China.

Combined transplantation of regulatory T cells (Treg cells) may significantly attenuate graft versus host disease (GVHD) after hematopoietic stem cell transplantation (HSCT). Recent studies indicated that CD150+Treg cells could secret adenosine to maintain the quiescent status of HSCs. However, whether it is attributable to the attenuation of GVHD after HSCT is still unclear. Read More

Bone Marrow Transplant 2019 Apr 10. Epub 2019 Apr 10.

Division of Hematology and Oncology, Department of Internal Medicine, The Jikei University School of Medicine, Tokyo, Japan.

Bone Marrow Transplant 2019 Apr 10. Epub 2019 Apr 10.

Departments of Medicine, Psychiatry, Clinical and Translational Science, University of Pittsburgh, Pittsburgh, PA, USA.

Understanding the potential emotional and psychological risks of pediatric sibling HSC donation is an area of research that remains in its infancy. A cross-sectional survey was distributed electronically to directors at all CIBMTR and EBMT centers to describe current transplant center practices for obtaining assent, preparation for the physical/emotional experiences of donation, and monitoring the post-donation well-being of pediatric donors (<18 years of age). Respondents were 45/91 (49%) and 66/144 (46%) of CIBMTR and EBMT centers, respectively. Read More

Indian J Pathol Microbiol 2019 Apr-Jun;62(2):279-282

Department of Transplant Immunology and Immunogenetics, All India Institute of Medical Sciences, New Delhi, India.

Griscelli syndrome is a rare autosomal recessive inherited disorder characterized by hypopigmentation, silver colored hair, and associated immunological deficiency, which proves fatal in the absence of timely intervention. Our patients diagnosed with Griscelli syndrome-2 presented with fever, hepatosplenomegaly, and deranged hematological and biochemical parameters. Both cases underwent detailed investigations comprising of hair mount microscopic examination, degranulation assay, and mutational studies. Read More