Trial reportBone marrow transplantation2007
Substitution of cyclophosphamide and busulfan by fludarabine, treosulfan and melphalan in a preparative regimen for children and adolescents with Shwachman-Diamond syndrome.
Trial report in Bone marrow transplantation, 2007. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 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
16 citing papers in PubMed, 46 citations in OpenAlex.
- Allogeneic Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for Treatment of Marrow Failure Disorders.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2017Trial
- Long-term follow-up of children conditioned with Treosulfan: German and Austrian experience.Bone marrow transplantation · 2013Trial
- Generation of NOD SCID mice with near-complete deletions of Il2rg and Prkdc for human cancer and HSC engraftment.Transgenic research · 2025Article
- Personalized hematopoietic stem cell transplantation for inborn errors of immunity.Frontiers in immunology · 2023Review
- Treosulfan-based conditioning for inborn errors of immunity.Therapeutic advances in hematology · 2021Review
- Long-term outcome after allogeneic hematopoietic stem cell transplantation for Shwachman-Diamond syndrome: a retrospective analysis and a review of the literature by the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation (SAAWP-EBMT).Bone marrow transplantation · 2020Review
- Hematopoietic Stem Cell Transplantation for Shwachman-Diamond Syndrome.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2020Article
- In Vitro Study of the Enzymatic and Nonenzymatic Conjugation of Treosulfan with Glutathione.European journal of drug metabolism and pharmacokinetics · 2019Article
- Allogeneic hematopoietic stem cell transplantation for inherited bone marrow failure syndromes.International journal of hematology · 2016Review
- Congenital neutropenia: diagnosis, molecular bases and patient management.Orphanet journal of rare diseases · 2011Review
- Human B cell development and antibody production in humanized NOD/SCID/IL-2Rγ(null) (NSG) mice conditioned by busulfan.Journal of clinical immunology · 2011Article
- Transplantation for congenital bone marrow failure syndromes.Bone marrow research · 2011Article
- Congenital disorders of ribosome biogenesis and bone marrow failure.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2010Review
- Shwachman-Diamond syndrome: a review of the clinical presentation, molecular pathogenesis, diagnosis, and treatment.Hematology/oncology clinics of North America · 2009Review
- Reduced-intensity conditioning is effective and safe for transplantation of patients with Shwachman-Diamond syndrome.Bone marrow transplantation · 2008Article
- Hematopoietic stem cell transplantation for childhood malignancies of myeloid origin.Bone marrow transplantation · 2008Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only definitive treatment for severe bone marrow dysfunction and clonal disorders in patients diagnosed with Shwachman-Diamond syndrome (SDS). In an attempt to minimize regimen-related toxicity (RRT), we have initiated a fludarabine/treosulfan/melphalan-based pilot protocol avoiding the combination of busulfan and cyclophosphamide. Median age at transplantation was 9.6 years (range 1.5-17 years). All three patients received conditioning with fludarabine (30 mg/m2/day x 6), treosulfan (12 g/m2/day x 3) and melphalan (140 mg/m2/day x 1). CAMPATH-1H (0.1 mg/kg x 2) was added in two cases, while rabbit ATG (Genzyme; 3 x 2.5 mg/kg) was given to the cord blood recipient. One patient was transplanted with a non-manipulated marrow graft from an HLA-identical sibling, one with a marrow graft from a 10/10 matched unrelated donor, and one with a 9/10 matched unrelated umbilical cord blood (UCB) unit. Mean cell doses given were 3.6 x 10(8) nucleated cells/kg BW for the bone marrow recipients and 4.2 x 10(7) nucleated cells/kg BW for UCB recipient. Overall, two of three patients are alive and display 100% donor chimerism. Acute graft-versus-host disease grade II was seen in one patient, while no GVHD exceeding grade I occurred in the remaining two.
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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.