Trial reportBone marrow transplantation2009
Allogeneic blood SCT for children with Hurler's syndrome: results from the German multicenter approach MPS-HCT 2005.
Trial report in Bone marrow transplantation, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
10 citing papers in PubMed, 23 citations in OpenAlex.
- Therapeutic potential of stem cells in pediatric neurology: Insights from clinical trials.Neuroprotection (Chichester, England) · 2025Review
- Predictors of growth patterns in children with mucopolysaccharidosis I after haematopoietic stem cell transplantation.JIMD reports · 2022Article
- Hematopoietic stem cell transplantation in mucopolysaccharidosis type IIIA: A case description and comparison with a genotype-matched control group.Molecular genetics and metabolism reports · 2020Article
- Considerations in Preparative Regimen Selection to Minimize Rejection in Pediatric Hematopoietic Transplantation in Non-Malignant Diseases.Frontiers in immunology · 2020Review
- Stem Cell Therapy for the Central Nervous System in Lysosomal Storage Diseases.Human gene therapy · 2016Review
- TCRαβ CD19 depletion in allogeneic haematopoietic stem cell transplantation performed for Hurler syndrome.Bone marrow transplantation · 2016Article
- Mesenchymal stem cells as cellular vectors for pediatric neurological disorders.Brain research · 2014Review
- Determination of intracellular fludarabine triphosphate in human peripheral blood mononuclear cells by LC-MS/MS.Journal of pharmaceutical and biomedical analysis · 2013Article
- Bone regeneration: stem cell therapies and clinical studies in orthopaedics and traumatology.Journal of cellular and molecular medicine · 2011Review
- Therapeutic liver repopulation for phenylketonuria.Journal of inherited metabolic disease · 2010Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
12 authors at 4 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Hurler's syndrome is an inborn error of mucopolysaccharide metabolism leading to premature death in childhood. Allogeneic hematopoietic SCT can achieve long-term survival by correcting the enzymatic deficiency. In an attempt to improve long-term engraftment and to reduce regimen-related toxicity (RRT), a prospective multicenter approach was initiated in Germany using a fludarabine-based radiation-free preparative regimen. Between 2001 and 2008, 12 children were enrolled. Median age at SCT was 14 months (range, 4-31 months). The conditioning regimen contained fludarabine, BU, melphalan and antithymocyte globulin. CD34 positively selected PBSC were used in 10 children with a matched unrelated donor. Median cell dose was 24.6 x 10(6) CD34+ cells per kg (range 10.0-54.8). Two children with a matched sibling donor received non-manipulated BM. Donor lymphocyte infusions were given in 6/12 children for mixed hematopoietic chimerism. At a median follow-up of 29 months (range 2-85 months), all children engrafted and have either stabilized or improved neurological function. In total, 12/12 patients showed donor-derived engraftment with 9/12 having full and 3/12 having mixed hematopoiesis. One developed acute GVHD >or=grade II. RRT >or=grade II was observed in two patients.
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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.