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Transplant Cell Ther 2021 Jun 9. Epub 2021 Jun 9.

Bone Marrow Transplant, Hospital Sirio Libanes, São Paulo, Brazil; Division of Hematology, Federal University of São Paulo (UNIFESP), São Paulo, Brazil.

Chronic graft-versus-host disease (cGVHD) remains a major barrier to a successful hematopoietic stem cell transplantation (HSCT); in cases refractory to first-line therapy with steroids, there is no standard of care for second-line therapy. As such, ruxolitinib is a promising drug in this scenario. We retrospectively analyzed its efficacy and safety as treatment of steroid-refractory cGVHD in 35 patients from two transplant centers, with the longest follow-up described to date. Read More

Autoimmun Rev 2021 Jun 9:102872. Epub 2021 Jun 9.

Department of Internal Medicine, Division of Allergy and Clinical Immunology, Erasmus Medical Center, Rotterdam, The Netherlands.

Objective: To describe the prevalence, clinical presentation and current treatment regimens of inflammatory bowel disease (IBD) in patients with primary immunodeficiency disorders (PIDs).

Methods: A systematic review was conducted. The following databases were searched: MEDLINE, Embase, Web of Science, the Cochrane Library and Google Scholar. Read More

Ann Hematol 2021 Jun 12. Epub 2021 Jun 12.

Department of Gerodontology and Oral Pathology, Poznan University of Medical Sciences, Bukowska 70, 60-812, Poznań, Poland.

This study was designed to investigate the frequency and severity of oral mucositis in patients with acute myeloid leukemia after allogeneic hematopoietic cell transplantation, in relation to the type of conditioning used. Eighty patients diagnosed with acute myeloid leukemia were assigned to two groups based on the conditioning regimen used before transplantation. The intensity of oral inflammatory lesions induced by chemotherapy (oral mucositis) was evaluated according to a 5-point scale recommended by World Health Organization. Read More

Cell Mol Immunol 2021 Jun 11. Epub 2021 Jun 11.

Université de Paris, Imagine Institute, Laboratory of Human Lymphohematopoiesis, INSERM UMR 1163, Paris, France.

Several obstacles to the production, expansion and genetic modification of immunotherapeutic T cells in vitro have restricted the widespread use of T-cell immunotherapy. In the context of HSCT, delayed naïve T-cell recovery contributes to poor outcomes. A novel approach to overcome the major limitations of both T-cell immunotherapy and HSCT would be to transplant human T-lymphoid progenitors (HTLPs), allowing reconstitution of a fully functional naïve T-cell pool in the patient thymus. Read More

Nat Commun 2021 Jun 11;12(1):3568. Epub 2021 Jun 11.

Division of Stem Cell Biology, Center for Stem Cell Biology and Regenerative Medicine, The Institute of Medical Science, The University of Tokyo, Tokyo, Japan.

Bone marrow (BM) chimeric mice are a valuable tool in the field of immunology, with the genetic manipulation of donor cells widely used to study gene function under physiological and pathological settings. To date, however, BM chimera protocols require myeloablative conditioning of recipient mice, which dramatically alters steady-state hematopoiesis. Additionally, most protocols use fluorescence-activated cell sorting (FACS) of hematopoietic stem/progenitor cells (HSPCs) for ex vivo genetic manipulation. Read More

J Immunother Cancer 2021 Jun;9(6)

Department. of Cellular Therapy and Allogeneic Stem Cell Transplantation, Karolinska University Hospital Huddinge, Karolinska Comprehensive Cancer Center, Stockholm, Sweden.

SARS-CoV-2 is the virus responsible for the COVID-19 pandemic. COVID-19 has highly variable disease severity and a bimodal course characterized by acute respiratory viral infection followed by hyperinflammation in a subset of patients with severe disease. This immune dysregulation is characterized by lymphocytopenia, elevated levels of plasma cytokines and proliferative and exhausted T cells, among other dysfunctional cell types. Read More

Am J Perinatol 2021 Jun 11. Epub 2021 Jun 11.

Division of Hematology and Oncology, Immunology, Department of Pediatrics, Erciyes University, School of Medicine, Kayseri, Turkey.

Objective:  T-cell receptor excision circles are expensive for neonatal severe combined immunodeficiency screening in developing countries. We aimed to detect immunodeficiencies presenting with lymphopenia to enable screening in the general population and to improve awareness regarding lymphopenia among clinicians.

Study Design:  This study was conducted prospectively. Read More

Blood 2021 Jun 11. Epub 2021 Jun 11.

Beijing Institute of Genomics, Chinese Academy of Sciences, China National Center for Bioinformation, Beijing, China.

Megakaryocytes (MKs), the platelet progenitor cell, play important roles in hematopoietic stem cell (HSC) maintenance and immunity. However, it is not known whether these diverse programs are executed by a single population or by distinct subsets of cells. Here, we manually-isolated primary CD41+ MKs from the bone marrow (BM) of mice and human donors based on ploidy (2N-32N), performed single-cell RNA sequencing analysis. Read More

OMICS 2021 Jun 25;25(6):342-357. Epub 2021 May 25.

International Center for Genetic Engineering and Biotechnology (ICGEB), Cape Town Component, Cape Town, South Africa.

Early cell biology reports demonstrated the presence of cells with stem-like properties in bone marrow, with both hematopoietic and mesenchymal lineages. Over the years, various investigations have purified and characterized mesenchymal stromal/stem cells (MSCs) from different human tissues as cells with multilineage differentiation potential under the appropriate conditions. Due to their appealing characteristics and versatile potentials, MSCs are leveraged in many applications in medicine such as oncology, bioprinting, and as recent as therapeutics discovery and innovation for COVID-19. Read More

Int J Paediatr Dent 2021 Jun 11. Epub 2021 Jun 11.

Instituto de Ensino e Pesquisa, Hospital Sírio-Libanês, São Paulo, Brazil.

Background: Oral mucositis (OM) is an important side effect related to allogeneic hematopoietic stem cell transplantation (allo-HSCT) and it has been associated with a significative reduction of quality of life. A negative impact of this toxicity in pediatric patients could result in an increased use of parenteral feeding and opioids, longer periods of hospitalization and higher risk of systemic infection.

Aim: To investigate the clinical features, and clinical outcomes associated with OM development and severity in hematologic cancer pediatric patients undergoing allo-HSCT who underwent professional dental care (PDC) and photobiomodulation (PBM) as prophylactic treatment. Read More

Eur J Haematol 2021 Jun 11. Epub 2021 Jun 11.

Clinical pharmacist/ clinical pharmacologist, Amsterdam University Medical Centers, department of Clinical Pharmacology and Pharmacy, Amsterdam, The Netherlands.

Objective: To investigate the incidence and severity of adverse drug reactions of cyclosporine using AUC targeted Therapeutic Drug Monitoring (TDM) compared to trough levels (C ) targeted TDM in adult allogeneic stem cell recipients.

Methods: Blind, monocentre, intervention study. Subjects were 1:1 randomized into either an AUC group or a C group. Read More

J Clin Immunol 2021 Jun 10. Epub 2021 Jun 10.

Department of Pediatric Immunology and Allergy, BMT Unit, Ankara University Medical School, Mamak Street, Dikimevi, 06100, Ankara, Turkey.

Severe combined immunodeficiency is an inborn error of immunity characterized by impairments in the numbers and functions of T and B lymphocytes due to various genetic causes, and if it remains untreated, patients succumb to infections during the first 2 years of life.

Purpose And Methods: This study reported retrospective data from 72 infants diagnosed with SCID including their major clinical features, HSCT characteristics, and outcomes over a 20-year period (1997-2017).

Results: Sixty-one of 72 SCID patients in the study underwent HSCT from 1997 to 2017. Read More

Front Pediatr 2021 25;9:678390. Epub 2021 May 25.

Department of Gastroenterology, Hepatology and Nutrition, Shanghai Children's Hospital, Shanghai Jiao Tong University, Shanghai, China.

Very early-onset inflammatory bowel disease (VEO-IBD) is defined as IBD diagnosed in children younger than 6 years of age. VEO-IBD is often associated with a monogenic etiology or primary immune deficiency. Here, we report the case of a 7-month-old Chinese girl diagnosed with VEO-IBD who had a variant in the interleukin-10 receptor A () gene. Read More

Immunohorizons 2021 Jun 10;5(6):424-437. Epub 2021 Jun 10.

Department of Hematology and Oncology, Okayama University Graduate School of Medicine, Dentistry and Pharmaceutical Sciences, Okayama, Japan

Graft-versus-host disease (GVHD) remains to be a significant cause of morbidity and mortality after allogeneic hematopoietic stem cell transplantation (HSCT). IL-2-inducible T cell kinase (ITK), a TEC cytoplasmic tyrosine kinase, has an essential role in T cell development and receptor signaling. The ITK/Bruton tyrosine kinase inhibitor ibrutinib has been shown to improve chronic GVHD symptoms; however, the effect of ITK selective inhibition on acute GVHD remains unclear. Read More

Blood Rev 2021 Jun 4:100853. Epub 2021 Jun 4.

Division of Hematology and Oncology, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, One Gustave L. Levi Place, Box 1079, New York, NY 10029, USA. Electronic address:

Hematopoietic stem cells (HSCs) have been used for therapeutic purposes for decades in the form of autologous and allogeneic transplantation and are currently emerging as an attractive target for gene therapy. A low stem cell dose is a major barrier to the application of HSC therapy in several situations, primarily umbilical cord blood transplantation and gene modification. Strategies that promote ex vivo expansion of the numbers of functional HSCs could overcome this barrier, hence have been the subject of intense and prolonged research. Read More

Stem Cell Res Ther 2021 Jun 10;12(1):336. Epub 2021 Jun 10.

Department of Pathology, Faculty of Veterinary Medicine, Cairo University, Giza, 12211, Egypt.

Background: Intra-bone marrow injection (IBMI) in rats is adopted in many studies for stem cell and hematopoietic cell transplantation. IBMI in the tibia or the femur results in severe distress to the animal. Therefore, this study aims to evaluate intra-iliac injections as an alternative approach for IBMI. Read More

Orphanet J Rare Dis 2021 Jun 10;16(1):269. Epub 2021 Jun 10.

Hematology Center, 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; Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, 100045, China.

Purpose: We intended to investigate the clinical features of paediatric patients with chronic active Epstein-Barr virus infection (CAEBV) and to examine the effectiveness of the L-DEP regimen before haematopoietic stem cell transplantation (HSCT).

Methods: A retrospective analysis was performed on 35 patients with CAEBV at Beijing Children's Hospital from January 2016 to January 2020. The efficacy and adverse events of the L-DEP regimen were evaluated. Read More

J Allergy Clin Immunol 2021 Jun 7. Epub 2021 Jun 7.

The Children's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.

Background: Heterozygous germline mutations in cytotoxic T-lymphocyte-associated antigen-4 (CTLA4) impair the immunomodulatory function of regulatory T cells. Affected individuals are prone to life-threatening autoimmune and lymphoproliferative complications. A number of therapeutic options are currently used with variable effectiveness. Read More

Am J Hematol 2021 Jun 10. Epub 2021 Jun 10.

Huntington Memorial Hospital, United States.

Introduction: Aggressive T-cell Lymphomas continue to have a poor prognosis. There are over 27 different subtypes of peripheral T-cell lymphoma (PTCL) and we are now beginning to understand the differences between the various subtypes beyond histologic variations.

Molecular Pathogenesis Of Various Subtypes Of Ptcl: Gene expression profiling (GEP) can help in diagnosis and prognostication of various subtypes including PTCL-nos and anaplastic large cell lymphoma (ALCL). Read More

Curr Treat Options Oncol 2021 Jun 10;22(8):65. Epub 2021 Jun 10.

Adults Hematology and Stem Cell Transplantation Department, King Fahad Specialist Hospital, 31444, Dammam, Kingdom of Saudi Arabia.

Opinion Statement: Nowadays, several novel agents have been introduced in the treatment of multiple myeloma, not only resulting in high response rates and prolonged survival but also offering good quality of life. However, the potential of cure, especially for patients with advanced or unfavorable disease features, remains elusive. Allogeneic hematopoietic stem cell transplantation, based mainly on the graft vs. Read More

Int J Immunogenet 2021 Jun 9. Epub 2021 Jun 9.

ZKRD - Zentrales Knochenmarkspender-Register für Deutschland, German National Bone Marrow Donor Registry, Ulm, Germany.

Unrelated haematopoietic stem cell transplantation (HSCT) has evolved from an experimental protocol to a potentially curative first-line treatment in a variety of haematologic malignancies. The continuous refinement of treatment protocols and supportive care paired with ongoing achievements in the technological field of histocompatibility testing enabled this transformation. Without a doubt, HLA matching is still the foremost criterion for donor selection in unrelated HSCT. Read More

Pediatr Blood Cancer 2021 Jun 9:e29174. Epub 2021 Jun 9.

Division of Hematologic Malignancies and Cellular Therapy, Duke Cancer Institute, Durham, North Carolina, USA.

Background: Limited English proficiency (LEP) is associated with adverse clinical outcomes. The clinical impact of LEP in hematopoietic stem cell transplant (HSCT) has not been studied. The objectives of this study were to compare HSCT outcomes and health care utilization of Hispanic pediatric patients with and without parental LEP. Read More

Bone Marrow Transplant 2021 Jun 9. Epub 2021 Jun 9.

IRCCS Azienda Ospedaliero-Universitaria di Bologna; Istituto "L. e A. Seràgnoli", Bologna, Italy.

Bone Marrow Transplant 2021 Jun 9. Epub 2021 Jun 9.

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

Two randomised trials (ASTIS, SCOT) of Autologous Hematopoietic Stem Cell Transplantation (AHSCT) versus monthly Cyclophosphamide for severe Systemic Sclerosis (SSc) patients used similar inclusion criteria, but different primary endpoints: event-free-survival (EFS) at 24 months in ASTIS versus the global rank composite score (GRCS) at 54 months in SCOT. Here we analysed the French ASTIS cohort (n = 49) outcome using the same GRCS endpoint as reported in SCOT. All patients, randomised to AHSCT (n = 26) or Cyclophosphamide (n = 23), were evaluated for the non-parametric GRCS endpoint based on: death, EFS, forced vital capacity (FVC), Health Assessment Questionnaire Disability Index (HAQ-DI) and modified Rodnan skin score (mRSS) at 60 months. Read More

Sci Rep 2021 Jun 9;11(1):12170. Epub 2021 Jun 9.

Department of Pediatric Hematology and Oncology, St. Anna Children's Hospital, Medical University of Vienna, Kinderspitalgasse 6, 1090, Vienna, Austria.

Acute appendicitis is a rare gastrointestinal complication of anti-cancer chemotherapy and hematopoietic stem cell transplantation. Among a cohort of 2341 hemato-oncologic patients at a pediatric tertiary care cancer center, we identified 21 patients (0.9%) with 23 episodes of acute appendicitis, based on pathological imaging of the appendix and clinical findings. Read More

Rinsho Ketsueki 2021 ;62(5):407-417

IMSUT Hospital, The Institute of Medical Science, The University of Tokyo.

The introduction of autologous stem cell transplantation, proteasome inhibitors, and immunomodulatory drugs (IMiDs) has improved the treatment outcome for multiple myeloma (MM). However, many patients develop resistance to existing therapies, and novel treatment strategies for these patients must be established. Therapeutic antibodies including daratumumab targeting CD38 and elotuzumab targeting SLAMF7 have been introduced in the clinic as immunotherapies for MM. Read More

Rinsho Ketsueki 2021 ;62(5):360-368

Department of Hematology, Shimane University Hospital.

Aggressive NK cell leukemia (ANKL) is a rare leukemic form of mature NK cell neoplasms. ANKL presents a fulminant clinical course with a median overall survival (OS) of 2-3 months after diagnosis. Currently, allogeneic stem cell transplantation (allo-HSCT) is the only curative treatment for ANKL patients. Read More

J Immunol 2021 Jun 9. Epub 2021 Jun 9.

Hematopoietic Stem Cell Transplantation and Primary Immune Deficiency, Department of Pediatrics and Adolescent Medicine, Copenhagen University Hospital Rigshospitalet, Copenhagen, Denmark.

Differentially and functionally distinct T cell subsets are involved in the development of complications after allogeneic hematopoietic stem cell transplantation (HSCT), but little is known about factors regulating their recovery after HSCT. In this study, we investigated associations between immune-regulating cytokines, T cell differentiation, and clinical outcomes. We included 80 children undergoing allogeneic HSCT for acute leukemia using bone marrow or peripheral blood stem cells grafted from a matched sibling or unrelated donor. Read More

Cancer Res 2021 Jun 9. Epub 2021 Jun 9.

Department of Medical Oncology, Dana-Farber Cancer Institute

The year 2020 marked the thirtieth anniversary of the Nobel Prize in Medicine awarded to E. Donnall Thomas for the development of allogeneic hematopoietic stem cell transplantation (allo-HSCT) to treat hematologic malignancies and other blood disorders. Dr. Read More

Ann Palliat Med 2021 May;10(5):5533-5540

State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.

Background: Cytomegalovirus (CMV) infection increases the risk of death after allogeneic hematopoietic stem cell transplantation (allo-HSCT). However, some patients do not respond to ganciclovir (GCV) or foscarnet sodium. Lyophilized intravenous human immunoglobulin containing high-titer CMV-neutralizing antibody (CMV-IVIG) is another option to further improve treatment safety and efficacy. Read More