Trial reportBone marrow transplantation2017
Changes in the incidence, patterns and outcomes of graft failure following hematopoietic stem cell transplantation for Hurler syndrome.
Trial report in Bone marrow transplantation, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05425043 (The Use of Granulocyte Transfusions After Umbilical Cord Blood Transplant for Leukaemia), which is not on this map. Cited by 24 papers.
What it found
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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.
The Use of Granulocyte Transfusions After Umbilical Cord Blood Transplant for Leukaemia: A Prospective, Non-randomised, Single-centre Study to Evaluate Safety and Immune Reconstitution
Who cites it
24 citing papers in PubMed, 40 citations in OpenAlex.
- α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Trial
- Reduced-intensity single-unit unrelated cord blood transplant with optional immune boost for nonmalignant disorders.Blood advances · 2020Trial
- Post-transplant laronidase augmentation for children with Hurler syndrome: biochemical outcomes.Scientific reports · 2019Trial
- Hematopoietic 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 transplantation · 2025Article
- Allogeneic hematopoietic stem cell transplantation for mucopolysaccharidosis patients: a single-center experience and assessment of quality of life.Italian journal of pediatrics · 2025Article
- Immune responses to cells and proteins after hematopoietic stem cell gene therapy for inherited diseases: A cause for concern.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Allogeneic stem cell transplantation for inherited metabolic disorders: 35 years' experience at a single institution.International journal of hematology · 2024Article
- Current and Future Treatment of Mucopolysaccharidosis (MPS) Type II: Is Brain-Targeted Stem Cell Gene Therapy the Solution for This Devastating Disorder?International journal of molecular sciences · 2022Review
- Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy.Blood advances · 2022Observational
- Strategies for Success With Umbilical Cord Haematopoietic Stem Cell Transplantation in Children With Malignant and Non-Malignant Disease Indications.Frontiers in cell and developmental biology · 2022Review
- The Outcome of Allogeneic Hematopoietic Stem Cell Transplantation From Different Donors in Recipients With Mucopolysaccharidosis.Frontiers in pediatrics · 2022Article
- Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease.Orphanet journal of rare diseases · 2021Article
- Hematopoietic stem cell transplantation in children with Griscelli syndrome type 2: a single-center report on 35 patients.Bone marrow transplantation · 2020Article
- Lower Exposure to Busulfan Allows for Stable Engraftment of Donor Hematopoietic Stem Cells in Children with Mucopolysaccharidosis Type I: A Case Report of Four Patients.International journal of molecular sciences · 2020Article
- A basic understanding of mucopolysaccharidosis: Incidence, clinical features, diagnosis, and management.Intractable & rare diseases research · 2020Review
- Considerations in Preparative Regimen Selection to Minimize Rejection in Pediatric Hematopoietic Transplantation in Non-Malignant Diseases.Frontiers in immunology · 2020Review
- Indications for haematopoietic stem cell transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe, 2019.Bone marrow transplantation · 2019Review
- Genetic testing of Mucopolysaccharidoses disease using multiplex PCR- based panels of STR markers: in silico analysis of novel mutations.Metabolic brain disease · 2019Article
- Review
- Hematopoietic Stem Cell Transplantation for Mucopolysaccharidoses: Past, Present, and Future.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2019Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors at 4 institutions in 2 countries.
Funding
Abstract
Hematopoietic stem cell transplantation (HSCT) is the standard of care in children with Hurler syndrome (HS) as it is the only therapy that can arrest disease progression. We examined the incidence, patterns and outcomes of graft failure in all HS children undergoing first HSCT at the Royal Manchester Children's Hospital or the University of Minnesota Children's Hospital from 1983 to 2016. Implementation of busulfan pharmacokinetic monitoring started in 2004 in both institutions. Two hundred and forty HS children were included in this analysis (historical era (pre-2004), n=131; current era (post 2004), n=109). The proportion of patients with graft failure was significantly lower in the current era compared with the historical era (37.2% vs 10.1%, respectively). Of 49 patients with graft failure in the historical era, 1 had aplasia and 48 had autologous reconstitution. All the 11 graft failures of the current era occurred in recipients of cord blood transplants (7 aplasia and 4 autologous reconstitution). The outcomes of second transplant in these patients has improved, with 89% of such patients alive and engrafted in the current era compared with 58% in the historical era. The pattern of graft failure has changed from autologous reconstitution, likely secondary to inadequate myelosuppression in the historical era, to aplasia in the current era, likely due to imperfect immunosuppression.
Indexed as
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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.