Trial reportBiology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation2016
Alternative-Donor Hematopoietic Stem Cell Transplantation with Post-Transplantation Cyclophosphamide for Nonmalignant Disorders.
Trial report in Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 35 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
35 citing papers in PubMed, 76 citations in OpenAlex.
- Feasibility of reduced-dose posttransplant cyclophosphamide and cotransplantation of peripheral blood stem cells and umbilical cord-derived mesenchymal stem cells for SAA.Scientific reports · 2021Trial
- Prospective Study of a Novel, Radiation-Free, Reduced-Intensity Bone Marrow Transplantation Platform for Primary Immunodeficiency Diseases.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2020Trial
- PTCy-based allo-BMT platform as a curative, accessible alternative for pediatric and young adult severe aplastic anemia.Blood advances · 2026Article
- Excellent Outcomes in Feasibility Study of Radiation-Free Conditioning and Transplant in Pediatric Severe Aplastic Anemia.Transplantation and cellular therapy · 2026Article
- Healthy volunteer stem cell donors followed up 10 years after stimulation with biosimilar filgrastim.Transfusion · 2025Article
- Haploidentical hematopoietic stem cell transplantation using post-transplant cyclophosphamide in patients with inborn errors of immunity: Experience in a reference center in ColombiaBiomedica : revista del Instituto Nacional de Salud · 2024Observational
- Haploidentical HSCT in the Treatment of Pediatric Hematological Disorders.International journal of molecular sciences · 2024Review
- Case report: HLA-haploidentical HSCT rescued with donor lymphocytes infusions in a patient with X-linked chronic granulomatous disease.Frontiers in immunology · 2023Article
- Case Report: The leopard sign as a potential characteristic of chronic granulomatous disease-associated colitis, unrelated to colitis severity.Frontiers in immunology · 2023Article
- Low-dose post-transplant cyclophosphamide with G-CSF/ATG based haploidentical protocol provides favorable outcomes for SAA patients.Frontiers in immunology · 2023Article
- HLA-haploidentical donor transplants with post-transplant cyclophosphamide in children with primary immune deficiency disorders.Bone marrow transplantation · 2022Article
- International retrospective study of allogeneic hematopoietic cell transplantation for activated PI3K-delta syndrome.The Journal of allergy and clinical immunology · 2022Article
- Haploidentical Hematopoietic Cell Transplantation Using Post-transplant Cyclophosphamide for Children with Non-malignant Diseases.Journal of clinical immunology · 2021Article
- Chronic Granulomatous Disease: a Comprehensive Review.Clinical reviews in allergy & immunology · 2021Review
- T-cell replete haploidentical transplantation with reduced post-transplant cyclophosphamide in six children with infantile osteopetrosis.Bone marrow transplantation · 2021Article
- Contemporary haploidentical stem cell transplant strategies in children with hematological malignancies.Bone marrow transplantation · 2021Review
- Assessing Early Supportive Care Needs among Son or Daughter Haploidentical Transplantation Donors.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2020Article
- Allogeneic hematopoietic stem cell transplantation using unrelated cord blood or unmanipulated haploidentical donors is effective in pediatric chronic granulomatous disease with inflammatory complications and severe infection.Bone marrow transplantation · 2020Article
- Article
- Incorporating posttransplant cyclophosphamide-based prophylaxis as standard-of-care outside the haploidentical setting: challenges and review of the literature.Bone marrow transplantation · 2020Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
13 authors at 2 institutions in 1 country.
Funding
Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) is curative for many nonmalignant pediatric disorders, including hemoglobinopathies, bone marrow failure syndromes, and immunodeficiencies. There is great success using HLA-matched related donors for these patients; however, the use of alternative donors has been associated with increased graft failure, graft-versus-host disease (GVHD), and transplant-related mortality (TRM). HSCT using alternative donors with post-transplantation cyclophosphamide (PT/Cy) for GVHD prophylaxis has been performed for hematologic malignancies with engraftment, GVHD, and TRM comparable with that seen with HLA-matched related donors. There are limited reports of HSCT in nonmalignant pediatric disorders other than hemoglobinopathies using alternative donors and PT/Cy. We transplanted 11 pediatric patients with life-threatening nonmalignant conditions using reduced-intensity conditioning, alternative donors, and PT/Cy alone or in combination with tacrolimus and mycophenolate mofetil. We observed limited GVHD, no TRM, and successful engraftment sufficient to eliminate manifestations of disease in all patients. Allogeneic HSCT using alternative donors and PT/Cy shows promise for curing nonmalignant disorders; development of prospective clinical trials to confirm these observations is warranted.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.