Allogeneic hematopoietic stem cell transplant (HSCT) has been known to be a curative therapy for patients with hemophagocytic lymphohistiocytosis (HLH) but donor availability is an issue. Haploidentical HSCT with posttransplant cyclophosphamide (PTCy) has been investigated as a feasible option for various malignant and nonmalignant conditions with reduced incidence of acute graft versus host disease (GVHD) and graft rejection. However, its use has not been described in children with HLH and here we describe 2 boys who underwent successful haploidentical HSCT with PTCy. None had acute GVHD and 1 had limited chronic GVHD. Both are alive and disease-free at follow-up of 912 and 239 days, respectively. Haploidentical HSCT with PTCy is a feasible option for children with HLH lacking a matched sibling donor.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1097/MPH.0000000000001265 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
The Combination of Intravenous Immunoglobulin, Dexamethasone, and a High Dose of Mononuclear Cells Transfusion: An Effective Strategy for Decreasing Donor-Specific Antibodies During Haploidentical Hematopoietic Stem Cell Transplantation.
Cell Transplant
January 2025
Department of Hematology, 920th Hospital of Joint Logistics Support Force, Kunming, China.
Donor-specific antibodies (DSAs) are essential causes of graft rejection in haploidentical hematopoietic stem cell transplantation (haplo-HSCT). DSAs are unavoidable for some patients who have no alternative donor. Effective interventions to reduce DSAs are still needed, and the cost of the current therapies is relatively high.
View Article and Find Full Text PDFInfluence of HLA-B Leader (-21M/T) Dimorphism With Bw4/Bw6 Epitopes on Graft Versus Host Disease After Allogenic Haematopoietic Stem Cell Transplantation in North Indians.
Int J Immunogenet
January 2025
Department of Clinical Haematology and Medical Oncology, Postgraduate Institute of Medical Education & Research, Chandigarh, India.
High degree of variability in human leukocyte antigens (HLAs) system restricts availability of histocompatible HLA-matched-related donors, thus increasing reliance on worldwide bone marrow registries network. Nevertheless, due to limited coverage/accessibility/affordability of some ethnicities in these registries, haploidentical haematopoietic stem cell transplantation (HSCT) emerged as an alternative option, though with allorecognition-mediated graft versus host disease (GvHD) (>40% cases). A dimorphism [-21 methionine (M) or threonine (T)] in HLA-B leader peptide (exon 1) which differentially influences its HLA-E binding, plausibly regulates natural killer cell functionality, affecting GvHD vulnerability and clinically in practice for donor selection.
View Article and Find Full Text PDFHematopoietic stem cell transplantation in inborn errors of metabolism-a retrospective analysis on behalf of the pediatric disease working party from the Brazilian Society of Bone Marrow Transplantation and Cellular Therapy.
Bone Marrow Transplant
January 2025
Instituto de Pesquisa Pelé Pequeno Príncipe/Faculdades Pequeno Príncipe, Curitiba, Brazil.
Hematopoietic stem cell transplantation (HSCT) is an established treatment for selected patients with inborn errors of metabolism. In this first report from the PDWP-SBTMO, we included 105 patients transplanted between 1988 and 2021 across six Brazilian HSCT centers. The most prevalent diseases were X-linked adrenoleukodystrophy (n = 61) and mucopolysaccharidosis (type I n = 20; type II n = 10), with a median age at HSCT of 8.
View Article and Find Full Text PDF[Hematopoietic Transplantation Outcomes in Patients with HLA-Identical, Haploidentical, and Unrelated Donors at a University Hospital in Chile].
Rev Med Chil
September 2024
Red UC Christus, Pontificia Universidad Católica de Chile, Santiago, Chile.
Unlabelled: Allogeneic transplantation (HSCT) is a curative option for several hematological diseases. Our center has privileged the use of identical family donors (IFD) or haploidentical (HD) donors. However, the chances of finding family donors may be challenging in small families or unsuitable donors.
View Article and Find Full Text PDFComparing haploidentical transplantation with post-transplantation cyclophosphamide and umbilical cord blood transplantation using targeted busulfan in children and adolescents with hematologic malignancies.
Blood Res
January 2025
Department of Pediatrics, Seoul National University College of Medicine, Seoul National University Cancer Research Institute, Seoul, Republic of Korea.
Purpose: This study compared the outcomes of haploidentical-related donor (HRD) and umbilical cord blood (UCB) hematopoietic stem cell transplantation (HSCT) in pediatric patients with hematologic malignancies.
Methods: Data on patients who underwent HRD HSCT with post-transplant cyclophosphamide (n = 41) and UCB HSCT (n = 24) after targeted busulfan-based myeloablative conditioning with intensive pharmacokinetic monitoring between 2009 and 2018 were retrospectively analyzed.
Results: The median follow-up durations in the HRD and UCB groups were 7.
Want AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!