63,445 results match your criteria Hematopoietic Stem Cell Transplantation


Reduced-intensity single-unit unrelated cord blood transplant with optional immune boost for nonmalignant disorders.

Blood Adv 2020 Jul;4(13):3041-3052

Division of Blood and Marrow Transplantation and Cellular Therapies and.

Children with many inherited nonmalignant disorders can be cured or their condition alleviated by hematopoietic stem cell transplantation (HSCT). Umbilical cord blood (UCB) units are a rapidly available stem cell source and offer great flexibility in HLA matching, allowing nearly uniform access to HSCT. Although reduced-intensity conditioning (RIC) regimens promise decreased treatment-related morbidity and mortality, graft failure and infections have limited their use in chemotherapy-naive patients. Read More

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http://dx.doi.org/10.1182/bloodadvances.2020001940DOI Listing

Efficient engraftment of genetically modified cells is necessary to ameliorate central nervous system involvement of murine model of mucopolysaccharidosis type II by hematopoietic stem cell targeted gene therapy.

Mol Genet Metab 2020 Jun 24. Epub 2020 Jun 24.

Division of Gene Therapy, Research Center for Medical Sciences, The Jikei University School of Medicine, Tokyo, Japan; Department of Pediatrics, The Jikei University School of Medicine, Tokyo, Japan. Electronic address:

Mucopolysaccharidosis type II (MPS II) is a lysosomal storage disease (LSD) caused by a deficiency of the iduronate-2-sulfatase (IDS) that catabolizes glycosaminoglycans (GAGs). Abnormal accumulations of GAGs in somatic cells lead to various manifestations including central nervous system (CNS) disease. Enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT) are the currently available therapy for MPS II, but both therapies fail to improve CNS manifestations. Read More

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http://dx.doi.org/10.1016/j.ymgme.2020.06.007DOI Listing

[Hematopoietic stem cell transplantation ocular complications: Guidelines from the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC)].

Bull Cancer 2020 Jul 3. Epub 2020 Jul 3.

CHU de Nantes, service d'hématologie, 1, place Alexis-Ricordeau, 44000 Nantes, France.

Introduction: Allogeneic stem cell transplantation is currently the only curative therapy for hematological disorders. This treatment can lead to complications, of which ophtalmological involvement.

Methods: We reviewed the literature and established accessible and convenient recommendations for hematologists and ophthalmologists. Read More

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http://dx.doi.org/10.1016/j.bulcan.2020.04.014DOI Listing

Pediatric Patients Treated for Leukemia Back to School: A Mixed-Method Analysis of Narratives about Daily Life and Illness Experience.

Behav Sci (Basel) 2020 Jul 1;10(7). Epub 2020 Jul 1.

Pediatric Hematology, Oncology and Stem Cell Transplant Center, Department of Woman's and Child's Health, University of Padua, 35128 Padua, Italy.

In the last few years, more children and adolescents healed from leukemia go back to their daily life, even if they can show some psycho-social difficulties. The study adopted semi-structured interviews and a mixed-method approach to examine the narratives of 75 children and adolescents about their return to school post 2-years treatment for leukemia. The aims are to collect their illness experiences, to understand how they feel about school and daily routines and to identify the best socio-demographic and illness predictors of a good re-adaptation to school and daily life. Read More

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http://dx.doi.org/10.3390/bs10070107DOI Listing

Nonsense Suppression Therapy: New Hypothesis for the Treatment of Inherited Bone Marrow Failure Syndromes.

Int J Mol Sci 2020 Jun 30;21(13). Epub 2020 Jun 30.

Cystic Fibrosis Center, Azienda Ospedaliera Universitaria Integrata, P.le A. Stefani 1, 37126 Verona, Italy.

Inherited bone marrow failure syndromes (IBMFS) are a group of cancer-prone genetic diseases characterized by hypocellular bone marrow with impairment in one or more hematopoietic lineages. The pathogenesis of IBMFS involves mutations in several genes which encode for proteins involved in DNA repair, telomere biology and ribosome biogenesis. The classical IBMFS include Shwachman-Diamond syndrome (SDS), Diamond-Blackfan anemia (DBA), Fanconi anemia (FA), dyskeratosis congenita (DC), and severe congenital neutropenia (SCN). Read More

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http://dx.doi.org/10.3390/ijms21134672DOI Listing

Severe COVID-19 in a patient with chronic graft-versus-host disease after hematopoietic stem cell transplant successfully treated with ruxolitinib.

Transpl Infect Dis 2020 Jul 6:e13401. Epub 2020 Jul 6.

Hematology and Stem Cell Transplant, Ancona University Hospital, Ancona, Italy.

Graft-versus-host disease (GVHD) is a common complication of hematopoietic stem cell transplant, which is known to be mediated by cytotoxic T-cell effectors and dysregulated inflammatory cytokines. Similarly, the lung injury observed in severe COVID-19 cases appears to be related to a massive production of proinflammatory cytokines. The selective JAK1/2 inhibitor ruxolitinib has shown promising results in the context of GVHD, and different trials are currently underway in patients with severe COVID-19; nevertheless no clinical observation of safety or efficacy of treatment with ruxolitinib in this context has been published yet. Read More

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http://dx.doi.org/10.1111/tid.13401DOI Listing

Hematopoietic stem-cell senescence and myocardial repair - Coronary artery disease genotype/phenotype analysis of post-MI myocardial regeneration response induced by CABG/CD133+ bone marrow hematopoietic stem cell treatment in RCT PERFECT Phase 3.

EBioMedicine 2020 Jul 3;57:102862. Epub 2020 Jul 3.

Reference and Translation Center for Cardiac Stem Cell Therapy, Department Life, Light and Matter and Department of cardiac surgery, University Medicine Rostock, Schillingallee 35, 18055 Rostock, Germany. Electronic address:

Background: Bone marrow stem cell clonal dysfunction by somatic mutation is suspected to affect post-infarction myocardial regeneration after coronary bypass surgery (CABG).

Methods: Transcriptome and variant expression analysis was studied in the phase 3 PERFECT trial post myocardial infarction CABG and CD133 bone marrow derived hematopoetic stem cells showing difference in left ventricular ejection fraction (∆LVEF) myocardial regeneration Responders (n=14; ∆LVEF +16% day 180/0) and Non-responders (n=9; ∆LVEF -1.1% day 180/0). Read More

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http://dx.doi.org/10.1016/j.ebiom.2020.102862DOI Listing

Low Non-Relapse Mortality after HLA Matched Related Two-Step Hematopoietic Stem Cell Transplantation using Cyclophosphamide (CY) for Graft versus Host Disease Prophylaxis and the Potential Impact of Non-CY-Exposed T Cells on Outcomes.

Biol Blood Marrow Transplant 2020 Jul 3. Epub 2020 Jul 3.

Department of Medical Oncology, Sidney Kimmel Cancer Center, Thomas Jefferson University.

The use of cyclophosphamide (CY) for bidirectional tolerization of recipient and donor T cells is associated with reduced rates of graft versus host disease (GVHD) and non-relapse mortality (NRM) after human leukocyte antigen (HLA) matched hematopoietic stem cell transplantation (HSCT). However, recurrent disease remains the primary barrier to long term survival. We extended our two-step approach to HLA matched related HSCT using a radiation-based myeloablative conditioning regimen combined with a high dose of T cells in an attempt to reduce relapse rates while maintaining the beneficial effects of CY tolerization. Read More

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http://dx.doi.org/10.1016/j.bbmt.2020.06.021DOI Listing

Autologous Hematopoietic Stem Cell Transplantation for Susac Syndrome.

Ann Intern Med 2020 Jul 7. Epub 2020 Jul 7.

Complejo Hospitalario Universitario de León, León, Spain (S.C., N.D.).

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http://dx.doi.org/10.7326/L20-0055DOI Listing

Factors Associated with Social Functioning Among Long-Term Cancer Survivors Treated with Hematopoietic Stem Cell Transplantation as Adolescents or Young Adults.

Psychooncology 2020 Jul 6. Epub 2020 Jul 6.

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

Objective: Hematopoietic stem cell transplantation (HSCT) can compromise long-term health and social functioning. We examined the impact of physical and social-emotional factors on the social functioning of long-term adolescent and young adult (AYA) HSCT survivors.

Methods: This cross-sectional analysis included HSCT recipients from the INSPIRE trial [NCT00799461] who received their first transplant between ages 15-39. Read More

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http://dx.doi.org/10.1002/pon.5460DOI Listing

Haploidentical Hematopoietic Stem Cell Transplantation for XIAP Deficiency: a Single-Center Report.

J Clin Immunol 2020 Jul 5. Epub 2020 Jul 5.

Beijing Key Laboratory of Pediatric Hematology Oncology; National Key Discipline of Pediatrics (Capital Medical University); Key Laboratory of Major Diseases in Children, Ministry of Education; Hematology Oncology Center, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, P.R., China.

Purpose: X-linked inhibitor of apoptosis (XIAP) deficiency caused by mutations in the XIAP/BIRC4 gene is a rare inherited primary immunodeficiency also known as X-linked lymphoproliferative syndrome type 2 (XLP2). Hematopoietic stem cell transplantation (HSCT) is currently the only curative strategy available. However, few studies of haploidentical HSCT have been published regarding the outcomes in patients with this syndrome. Read More

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http://dx.doi.org/10.1007/s10875-020-00795-6DOI Listing

Long-Term Remission Achieved by Ponatinib and Donor Lymphocytes Infusion in a Ph+ Acute Lymphoblastic Leukemia Patient in Molecular Relapse After Allogenic Stem Cell Transplant and Dasatinib: A Case Report.

Front Oncol 2020 18;10:967. Epub 2020 Jun 18.

Hematology Unit, Hematology and Oncology Department, "Annunziata" Hospital of Cosenza, Cosenza, Italy.

Currently, the prognosis of Ph+ acute lymphoblastic leukemia (Ph+ ALL) patients relapsing after an allogenic hematopoietic stem cell transplantation (allo-SCT) remains poor, with few therapeutic options available. Here we present the case of a 32 years old patient with dasatinib-resistant post-transplant molecular relapse of ALL, who received, as second-line therapy, the combination of ponatinib and donor lymphocyte infusion (DLI). The therapy was safe and the patient achieved a sustained minimal residual disease negative disease, still ongoing after 22 months, which was accompanied by several changes in the immune populations distribution within the bone marrow (i. Read More

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http://dx.doi.org/10.3389/fonc.2020.00967DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7314974PMC

Phase I dose escalation study of naive T-cell depleted donor lymphocyte infusion following allogeneic stem cell transplantation.

Bone Marrow Transplant 2020 Jul 5. Epub 2020 Jul 5.

Division of Hematologic Malignancies and Cellular Therapy, Duke University Medical Center, Durham, NC, USA.

Prophylactic donor lymphocyte infusions (DLI) are used to augment post-transplant immune recovery to reduce both infectious complications and disease recurrence. Preclinical studies implicate the naive T-cell subset as the primary driver of graft-versus-host disease (GvHD). In this phase I dose escalation study, we assessed the safety of a DLI that was depleted of CD45RA+ naive T cells. Read More

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http://dx.doi.org/10.1038/s41409-020-0991-5DOI Listing

[Challenges of screening germline predispositions in children].

Authors:
Atsushi Manabe

Rinsho Ketsueki 2020 ;61(6):682-686

Department of Pediatrics, Hokkaido University Graduate School of Medicine.

Genetic predisposition is a major cause of childhood cancer. Multiple cancer-predisposing syndromes have been identified, including Li-Fraumeni syndrome (LFS), neurofibromatosis type 1, APC-related adenomatous polyposis, Beckwith-Wiedemann syndrome, multiple endocrine neoplasia 1, ataxia telangiectasia, RUNX1 deficiency, Fanconi anemia, Bloom syndrome, and PTEN hamartoma tumor syndrome. LFS is a prototypical genetically predisposing condition. Read More

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http://dx.doi.org/10.11406/rinketsu.61.682DOI Listing
January 2020

[Graft-versus-host disease targets R-spondin3-producing lymphatic endothelial cells in the small intestine].

Authors:
Reiki Ogasawara

Rinsho Ketsueki 2020 ;61(6):621-627

Department of Hematology, Hokkaido University Faculty of Medicine and Graduate School of Medicine.

Graft-versus-host disease (GVHD) is a potentially fatal complication of allogeneic hematopoietic stem cell transplantation. The gastrointestinal tract is a major target organ of GVHD, and disruption of the barrier function of the intestinal mucosa leads to an influx of danger signals derived from intestinal microbiota, which may further exaggerate GVHD. We have shown that the recombinant human R-spondin1 protects intestinal stem cells against GVHD, improves intestinal dysbiosis, and ameliorates GVHD. Read More

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http://dx.doi.org/10.11406/rinketsu.61.621DOI Listing
January 2020

[COVID-19 and hematopoietic stem cell transplantation: Recommendations].

Bull Cancer 2020 Jun 20. Epub 2020 Jun 20.

Mohammed VI University of Health Sciences (UM6SS), Cheick-Khalifa International University Hospital, Department of Medical Oncology, Casablanca, Maroc. Electronic address:

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http://dx.doi.org/10.1016/j.bulcan.2020.05.006DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7305863PMC

Effect of Sirolimus levels between days 11 and 20 after allogeneic stem cell transplantation on the risk of hepatic sinusoidal obstruction syndrome.

Bone Marrow Transplant 2020 Jul 4. Epub 2020 Jul 4.

Department of Medicine, Division of Hematology and Oncology, Indiana University School of Medicine, Indianapolis, IN, USA.

Sinusoidal obstruction syndrome (SOS) is a serious complication of hematopoietic stem cell transplantation (HSCT). Sirolimus plus tacrolimus is an accepted regimen for graft-versus-host disease (GVHD) prophylaxis, with both agents implicated as risk factors for SOS. We analyzed 260 consecutive patients who underwent allogeneic HSCT following myeloablative conditioning using total body irradiation (TBI)-based (n = 151) or chemotherapy only (n = 109) regimens, with sirolimus plus tacrolimus for GVHD prophylaxis. Read More

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http://dx.doi.org/10.1038/s41409-020-0987-1DOI Listing

Gene Therapy for Wiskott-Aldrich Syndrome: History, New Vectors, Future Directions.

J Allergy Clin Immunol 2020 Jul 2. Epub 2020 Jul 2.

Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics and Institute for Stem Cell Biology and Regenerative Medicine, Stanford School of Medicine, Stanford, CA, United States. Electronic address:

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http://dx.doi.org/10.1016/j.jaci.2020.06.018DOI Listing

Long-term outcomes of innovator versus generic melphalan formulation in autologous hematopoietic cell transplantation for multiple myeloma.

Hematol Oncol Stem Cell Ther 2020 Jun 25. Epub 2020 Jun 25.

Department of Internal Medicine, Postgraduate Institute of Medical Education and Research, Chandigarh, India.

Background: Most data on autologous hematopoietic cell transplantation (auto-HCT) in myeloma are based on the use of innovator formulation of melphalan. Comparative bioequivalence and efficacy studies of generic melphalan are lacking.

Methods: In this retrospective study, we report long-term outcomes of auto-HCT in myeloma using innovator (Alkeran, Aspen Pharma; n = 41) and generic melphalan (Alkacel, Celon Labs, India; n = 55) formulations. Read More

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http://dx.doi.org/10.1016/j.hemonc.2020.06.002DOI Listing

Early CD4+ T cell reconstitution as predictor of outcomes after allogeneic hematopoietic cell transplantation.

Cytotherapy 2020 Jul 1. Epub 2020 Jul 1.

Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York, New York, USA.

Background: An association between early CD4+ T cell immune reconstitution (CD4+ IR) and survival after T-replete allogeneic hematopoietic cell transplantation (HCT) has been previously reported. Here we report validation of this relationship in a separate cohort that included recipients of ex vivo T-cell-depleted (TCD) HCT. We studied the relationship between CD4+ IR and clinical outcomes. Read More

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http://dx.doi.org/10.1016/j.jcyt.2020.05.005DOI Listing

Regulation of ABO blood group antigen expression by miR-331-3p and miR-1908-5p during hematopoietic stem cell differentiation.

Stem Cells 2020 Jul 4. Epub 2020 Jul 4.

Department of Experimental Transfusion Medicine, Medical Faculty Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.

The ABO blood group system is the most important factor in clinical transfusion medicine and is implicated in a number of human diseases. ABO antigens are not confined to red blood cells (RBCs) and are widely expressed in a variety of human cells and tissues. To date, many alleles with variant ABO expression have been identified and in many cases trace to one of the >250 reported genetic variations in the respective glycosyltransferase. Read More

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http://dx.doi.org/10.1002/stem.3251DOI Listing

Lymphoma Predisposing Gene in an Extended Family: CD70 Signaling Defect.

J Clin Immunol 2020 Jul 3. Epub 2020 Jul 3.

Aziz Sancar Institute of Experimental Medicine, Genetics Department, Istanbul University, Istanbul, Turkey.

Genome-wide sequencing studies in pediatric cancer cohorts indicate that about 10% of patients have germline mutations within cancer predisposition genes. Within this group, primary immune deficiencies take the priority regarding the vulnerability of the patients to infectious agents and the difficulties of cancer management. On the other hand, early recognition of these diseases may offer specific targeted therapies and hematopoietic stem cell transplantation as an option. Read More

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http://dx.doi.org/10.1007/s10875-020-00816-4DOI Listing

Optimal pre-emptive cytomegalovirus therapy threshold in a patient population with high prevalence of seropositive status.

Curr Res Transl Med 2020 Jun 30. Epub 2020 Jun 30.

Division of Stem Cell Transplantation and Cellular Therapy, Department of Oncology, King Abdulaziz Medical City, Riyadh, KSA, Saudi Arabia; King Abdullah International Medical Research Center, Riyadh, KSA, Saudi Arabia; King Saud Bin Abdulaziz University for Health Sciences, Riyadh, KSA, Saudi Arabia.

Introduction: Preemptive therapy (PET) for cytomegalovirus (CMV) reactivation post allogeneic hematopoietic stem cell transplantation (SCT) was shown to decrease the incidence of CMV disease. However, the optimal PET threshold is elusive.

Purpose Of The Study: To examine the efficacy of PET initiation at a viral threshold of 1000 copies/mL (1560 IU/mL) in a patient population with high prevalence of CMV seropositive status. Read More

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http://dx.doi.org/10.1016/j.retram.2020.04.005DOI Listing

Impact of CD44 gene single nucleotide polymorphism (rs 13347) in mobilization of autologous HSCT.

Transfus Apher Sci 2020 Jun 27:102869. Epub 2020 Jun 27.

Department of Internal Medicine, Faculty of Medicine, Alexandria University, Egypt.

MOBILIZATION: of stem cells into peripheral blood is a crucial step in the procedure of autologous stem cell transplantation. Mobilization can be affected by many variables;underlying diseases, prior treatment and age. Many genetic polymorphisms mainly in adhesion molecules are thought to affect mobilization success. Read More

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http://dx.doi.org/10.1016/j.transci.2020.102869DOI Listing

Pituitary Apoplexy During Hematopoietic Cell Transplantation.

Clin Lymphoma Myeloma Leuk 2020 May 26. Epub 2020 May 26.

Department of Hematology-Oncology, Vanderbilt University Medical Center, Nashville, TN. Electronic address:

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http://dx.doi.org/10.1016/j.clml.2020.05.015DOI Listing

[Intensification by high dose chemotherapy for germ-cell tumors, still an ongoing subject?]

Bull Cancer 2020 Jun;107(5S):S41-S48

Département de cancérologie médicale, centre Léon-Bérard, 28, rue Laennec, 69008 Lyon, France.

More than 80 % of patient with metastatic germ cell tumor are cured with first line chemotherapy. Twenty to 30 % of patients will experience relapse or refractory disease with a very poor long-term prognosis. Most of them had metastatic germ cell tumors with a poor prognosis according to the international germ cell classification collaborative group (IGCCCG). Read More

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http://dx.doi.org/10.1016/S0007-4551(20)30277-0DOI Listing

Design of a Randomized Placebo Controlled Trial of High Dose Intravenous Thiamine for the Prevention of Delirium in Allogeneic Hematopoietic Stem Cell Transplantation.

Contemp Clin Trials 2020 Jun 30:106076. Epub 2020 Jun 30.

Department of Psychiatry, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA; Lineberger Comprehensive Cancer Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA; Department of Medicine, Division of Hematology/Oncology, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.

Background: Delirium is a highly prevalent and preventable neuropsychiatric condition with major health consequences. Thiamine deficiency is a well-established cause of delirium in those with chronic, severe alcoholism, but there remains an underappreciation of its significance in non-alcoholic populations, including patients with cancer. Treatment of suspected thiamine-related mental status changes with high dose intravenous (IV) thiamine has preliminary evidence for improving a variety of cognitive symptoms in oncology inpatient settings but has never been studied for the prevention of delirium in any population. Read More

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http://dx.doi.org/10.1016/j.cct.2020.106076DOI Listing

Curative outcomes following blinatumomab in adults with minimal residual disease B-cell precursor acute lymphoblastic leukemia.

Leuk Lymphoma 2020 Jul 3:1-9. Epub 2020 Jul 3.

Comprehensive Cancer Center Mainfranken, Uniklinikum Würzburg, Würzburg, Germany.

Minimal residual disease (MRD) is the strongest predictor of relapse in B-cell precursor acute lymphoblastic leukemia (BCP-ALL). In BLAST study (NCT01207388), adults with BCP-ALL in remission with MRD after chemotherapy received blinatumomab, a CD19 BiTE immuno-oncotherapy, 15 µg/m/day for up to four 6-week cycles (4 weeks continuous infusion, 2 weeks off). Survival was evaluated for 110 patients, including 74 who received HSCT in continuous complete remission. Read More

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http://dx.doi.org/10.1080/10428194.2020.1780583DOI Listing

Aplastic anaemia: Current concepts in diagnosis and management.

J Paediatr Child Health 2020 Jul 3. Epub 2020 Jul 3.

Department of Paediatric and Adolescent Haematology, Oncology, Blood and Marrow Transplantation, Perth Children's Hospital, Perth, Western Australia, Australia.

Aplastic anaemia is a rare, previously fatal condition with a significantly improved survival rate owing to advances in understanding of the pathophysiology and improved treatment strategies including haematopoietic stem cell transplantation. Although a rare condition, aplastic anaemia continues to present a high burden for affected patients, their families and the health system due to the prolonged course of disease often associated with high morbidity and the uncertainty regarding clinical outcome. Modern molecular and genetic techniques including next-generation sequencing have contributed to a better understanding of this heterogeneous group of conditions, albeit at a cost of increased complexity of clinical decision-making regarding prognosis and choice of treatment for individual patients. Read More

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http://dx.doi.org/10.1111/jpc.14996DOI Listing

Senescence-associated secretory phenotype promotes chronic ocular graft-vs-host disease in mice and humans.

FASEB J 2020 Jul 3. Epub 2020 Jul 3.

Department of Ophthalmology, Keio University School of Medicine, Tokyo, Japan.

Chronic graft-vs-host disease (cGVHD) is a multifactorial inflammatory disease that affects patients undergoing hematopoietic stem cell transplantation. Multiple organs, including the lacrimal glands (LGs), are negatively affected by cGVHD and lose function due to the resultant fibrosis. An abnormal immune response is thought to be a major factor in the development of chronic ocular GVHD, which is currently treated primarily with immunosuppressive therapies. Read More

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http://dx.doi.org/10.1096/fj.201900218RDOI Listing

Haploidentical Transplants for Patients with Relapse After the First Allograft.

Am J Hematol 2020 Jul 3. Epub 2020 Jul 3.

Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.

Relapse after allogeneic stem-cell transplantation (AHSCT) is associated with very poor outcomes. A second transplant offers the possibility of long-term disease control. We analyzed outcomes with haploidentical donors for second allograft at our institution. Read More

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http://dx.doi.org/10.1002/ajh.25924DOI Listing

Risk factors for mixed chimerism in children with hemophagocytic lymphohistiocytosis after reduced toxicity conditioning.

Pediatr Blood Cancer 2020 Jul 3:e28523. Epub 2020 Jul 3.

Division of Pediatric Stem Cell Transplantation and Immunology, University Medical Center Hamburg, Hamburg, Germany.

Background: Reduced toxicity conditioning for hematopoietic stem cell transplantation of patients with hemophagocyticlymphohistiocytosis (HLH) results in favorable survival, however at the expense of relevant rates of mixed chimerism. Factors predisposing to mixed chimerism remain to be determined.

Procedure: Patients with primary HLH transplanted 2009-2016 after treosulfan- or melphalan-based conditioning regimens were analyzed in a retrospective multicenter study for survival, engraftment, chimerism, and adverse events. Read More

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http://dx.doi.org/10.1002/pbc.28523DOI Listing

Hematopoietic stem cell transplantation for people with sickle cell disease.

Cochrane Database Syst Rev 2020 Jul 3;7:CD007001. Epub 2020 Jul 3.

Paediatrics Department, National Hospital, Abuja, Nigeria.

Background: Sickle cell disease is a genetic disorder involving a defect in the red blood cells due to its sickled hemoglobin. The main therapeutic interventions include preventive and supportive measures. Hematopoietic stem cell transplantations are carried out with the aim of replacing the defective cells and their progenitors (hematopoietic (i. Read More

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http://dx.doi.org/10.1002/14651858.CD007001.pub5DOI Listing

A 23-Year Follow-Up of a Patient with Gain-of-Function IkB-Alpha Mutation and Stable Full Chimerism After Hematopoietic Stem Cell Transplantation.

J Clin Immunol 2020 Jul 2. Epub 2020 Jul 2.

University Department of Pediatrics, Unit of Immune and Infectious Diseases, Childrens' Hospital Bambino Gesù, Piazza S. Onofrio 4, Rome, Italy.

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http://dx.doi.org/10.1007/s10875-020-00780-zDOI Listing

Neurocognitive benchmarks following transplant for emerging cerebral adrenoleukodystrophy.

Neurology 2020 Jul 2. Epub 2020 Jul 2.

From the Departments of Pediatrics (E.I.P., R.S.Z., W.P.M., A.O.G., T.C.L., P.J.O., J.B.E.), Radiology (D.R.N.), Bioinformatics and Biostatistics Core (R.S.), and Neurology (D.L.K.-J.), University of Minnesota, Minneapolis; and Sangamo Therapeutics (W.P.M.), Richmond, CA.

Objective: To quantify benchmark treatment outcomes that may be enabled by newborn screening surveillance for X-linked adrenoleukodystrophy (ALD), we report neurocognitive, neuropsychiatric, and MRI change for boys who underwent hematopoietic stem cell transplant (HSCT) at initial stages of demyelination, prior to neurocognitive signs of disease.

Methods: Retrospective chart review identified 36 patients whose cerebral ALD was detected and treated early, with lesion severity less than 5 on the ALD-specific MRI scoring system. Median age at transplant was 7. Read More

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http://dx.doi.org/10.1212/WNL.0000000000009929DOI Listing

Anti-PD-1 Therapy Associated Type 1 Diabetes in a Pediatric Patient With Relapsed Classical Hodgkin Lymphoma.

Diabetes Care 2020 Jul 2. Epub 2020 Jul 2.

Department of Diabetes Immunology, Diabetes & Metabolism Research Institute, City of Hope, Duarte, CA

Objective: Immune checkpoint inhibitors (ICIs) perturb T-cell regulatory pathways to enhance antitumor immunity. However, an increase reporting of ICI-associated diabetes is observed in adults. To our knowledge, no cases have been reported in the pediatric population. Read More

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http://dx.doi.org/10.2337/dc20-0740DOI Listing

Etoposide-mediated interleukin-8 secretion from bone marrow stromal cells induces hematopoietic stem cell mobilization.

BMC Cancer 2020 Jul 2;20(1):619. Epub 2020 Jul 2.

Division of Hematology-Oncology, Department of Internal Medicine, Korea University School of Medicine, 73, Goryeodae-ro, Seongbuk-gu, Seoul, 02841, South Korea.

Background: We assessed the mechanism of hematopoietic stem cell (HSC) mobilization using etoposide with granulocyte-colony stimulating factor (G-CSF), and determined how this mechanism differs from that induced by cyclophosphamide with G-CSF or G-CSF alone.

Methods: We compared the clinical features of 173 non-Hodgkin's lymphoma patients who underwent autologous peripheral blood stem cell transplantation (auto-PBSCT). Additionally, we performed in vitro experiments to assess the changes in human bone marrow stromal cells (hBMSCs), which support the HSCs in the bone marrow (BM) niche, following cyclophosphamide or etoposide exposure. Read More

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http://dx.doi.org/10.1186/s12885-020-07102-xDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7330970PMC

[Hepatolienal candidosis as a rare differential diagnosis of disseminated small parenchym lesions].

Dtsch Med Wochenschr 2020 Jul 2;145(13):912-916. Epub 2020 Jul 2.

Klinik für Diagnostische und Interventionelle Radiologie, Uniklinikum Ulm, Ulm.

History:  We report about a 17-year-old patient with the secondary malignancy of acute myeloid leukemia (AML). He developed fever of unclear origin during the hematopoietic stem cell transplantation.History We report about a 17-year-old patient with the secondary malignancy of acute myeloid leukemia (AML). Read More

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http://dx.doi.org/10.1055/a-1164-0043DOI Listing

Inflammation-driven activation of JAK/STAT signaling reversibly accelerates acute myeloid leukemia in vitro.

Blood Adv 2020 Jul;4(13):3000-3010

Department of Hematology, University Hospital Essen, Essen, Germany.

Acute myeloid leukemia (AML) is characterized by a high relapse rate and dismal long-term overall survival which is related to persistence of leukemia-initiating cells in their niche. Different animal models of myeloid malignancies reveal how neoplastic cells alter the structural and functional characteristics of the hematopoietic stem cell niche to reinforce malignancy. Understanding and disruption of the microenvironmental interactions with AML cells are a vital need. Read More

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http://dx.doi.org/10.1182/bloodadvances.2019001292DOI Listing

Risk stratification and outcomes of intracranial hemorrhage in patients with immune thrombocytopenia under 60 years of age.

Platelets 2020 Jul 2:1-9. Epub 2020 Jul 2.

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

Intracranial hemorrhage (ICH) is a devastating complication of immune thrombocytopenia (ITP). However, information on ICH in ITP patients under the age of 60 years is limited, and no predictive tools are available in clinical practice. A total of 93 adult patients with ITP who developed ICH before 60 years of age were retrospectively identified from 2005 to 2019 by 27 centers in China. Read More

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http://dx.doi.org/10.1080/09537104.2020.1786042DOI Listing

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

Pediatr Blood Cancer 2020 Jul 2:e28233. Epub 2020 Jul 2.

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

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

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

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http://dx.doi.org/10.1002/pbc.28233DOI Listing

Whole-Genome Approach to Assessing Human Cytomegalovirus Dynamics in Transplant Patients Undergoing Antiviral Therapy.

Front Cell Infect Microbiol 2020 15;10:267. Epub 2020 Jun 15.

MRC-University of Glasgow Centre for Virus Research, Glasgow, United Kingdom.

Human cytomegalovirus (HCMV) is the most frequent cause of opportunistic viral infection following transplantation. Viral factors of potential clinical importance include the selection of mutants resistant to antiviral drugs and the occurrence of infections involving multiple HCMV strains. These factors are typically addressed by analyzing relevant HCMV genes by PCR and Sanger sequencing, which involves independent assays of limited sensitivity. Read More

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http://dx.doi.org/10.3389/fcimb.2020.00267DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7308726PMC

First case of near haploid philadelphia negative B-Cell acute lymphoblastic leukaemia relapsing as acute myeloid leukemia following allogeneic hematopoietic stem cell transplantation.

Leuk Res Rep 2020 18;14:100213. Epub 2020 Jun 18.

University Hospitals Birmingham NHS Foundation Trust, Heartlands Hospital.

Herein we present a female patient aged 61 with Philadelphia negative acute lymphoblastic leukaemia demonstrating near haploid karyotype and abnormal TP53 expression at diagnosis, who relapsed with lineage switch as Acute Monocytic Leukemia post allogeneic stem cell transplantation. Molecular analysis established that both neoplasms were derived from the same founder clone. The leukemic lineage switch phenomenon has recently re-attracted interest as mechanism of leukemic evasion post treatment with chimeric antigen receptor T-cells but there is paucity of data on its presence post allograft or following novel antibody treatments such as Inotuzumab Ozogamicin or Blinatumomab. Read More

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http://dx.doi.org/10.1016/j.lrr.2020.100213DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7317226PMC

Management of central nervous system involvement in multiple myeloma after autologous hematopoietic stem cell transplantation.

Leuk Res Rep 2020 10;14:100210. Epub 2020 Jun 10.

Hematology, Oncology and Stem Cell Transplantation Research Center, Shariati Hospital, Tehran University of Medical Sciences, Tehran, Iran.

Multiple myeloma (MM) is characterized by uncontrolled clonal proliferation of plasma cells, mainly in bone marrow, and its extramedullary involvement is rare. Central nervous system involvement in MM is a highly aggressive disease with a survival of less than 6 months. The best treatment regimen for MM with CNS involvement is still unknown and in most patients, the prognosis is unfavorable. Read More

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http://dx.doi.org/10.1016/j.lrr.2020.100210DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7317678PMC

Cardiac safe hematopoietic stem cell transplantation for systemic sclerosis with poor cardiac function: a pilot safety study that decreases neutropenic interval to 5 days.

Bone Marrow Transplant 2020 Jul 1. Epub 2020 Jul 1.

Department of Preventive Medicine, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.

We compared three fludarabine-based regimens for systemic sclerosis patients with a high-risk cardiac phenotype that according to EBMT criteria would be a contraindication for a high-dose cyclophosphamide (200 mg/kg) transplant regimen. All three regimens included fludarabine, ATG, and cyclophosphamide (60 mg/kg), while two regimens also included rituximab with or without IVIG. Treatment related mortality (TRM) was 2. Read More

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http://dx.doi.org/10.1038/s41409-020-0978-2DOI Listing

Localized Lymph Node Light Chain Amyloidosis.

Intern Med 2020 Jun 30. Epub 2020 Jun 30.

Department of General Medicine, Okayama University Graduate School of Medicine, Dentistry and Pharmaceutical Sciences, Japan.

The prognosis of systemic amyloid light chain (AL) amyloidosis is generally poor, hence requiring chemotherapy or hematopoietic stem cell transplantation, while the prognosis of localized AL amyloidosis without an abnormal monoclonal immunoglobulin light chain is good. Localized AL amyloidosis has been previously reported to be observed in pulmonary, urinary tract, gastrointestinal, oropharyngeal, and laryngeal sites. However, only a few cases of localized lymph node AL amyloidosis have so far been reported. Read More

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http://dx.doi.org/10.2169/internalmedicine.4651-20DOI Listing

Intestinal goblet cells protect against GVHD after allogeneic stem cell transplantation via Lypd8.

Sci Transl Med 2020 Jul;12(550)

Department of Hematology, Hokkaido University Faculty of Medicine and Graduate School of Medicine, Sapporo 060-8638, Japan.

Graft-versus-host disease (GVHD) and infection are major obstacles to successful allogeneic hematopoietic stem cell transplantation (HSCT). Intestinal goblet cells form the mucus layers, which spatially segregate gut microbiota from host tissues. Although it is well known that goblet cell loss is one of the histologic features of GVHD, effects of their loss in pathophysiology of GVHD remain to be elucidated. Read More

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http://dx.doi.org/10.1126/scitranslmed.aaw0720DOI Listing

Immune responses to hepatitis B vaccination after hematopoietic stem cell transplantation in pediatric and young adult patients.

Clin Transplant 2020 Jul 1:e14024. Epub 2020 Jul 1.

Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital, Mahidol University, 270 Rama VI Road, Bangkok, 10400, Thailand.

Background: Hematopoietic stem cell transplantation (HSCT) recipients require hepatitis B (HBV) revaccination. Hepatitis B surface antibody (anti-HBs) seroconversion rates after revaccination range from 64%-79% in these patients. The seroconversion rate and factors associated with non-seroconversion have not been clearly elucidated in pediatric and young adult recipients after HSCT. Read More

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http://dx.doi.org/10.1111/ctr.14024DOI Listing