Trial reportBone marrow transplantation2006
Stem cell transplantation in children with infantile osteopetrosis is associated with a high incidence of VOD, which could be prevented with defibrotide.
Trial report in Bone marrow transplantation, 2006. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 38 papers, 2 of them syntheses that pooled it.
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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
38 citing papers in PubMed, 2 syntheses or guidelines pooled it, 106 citations in OpenAlex.
- A Systematic Review and Meta-Analysis of Studies of Defibrotide Prophylaxis for Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome.Clinical drug investigation · 2022Pooled it
- Interventions for prophylaxis of hepatic veno-occlusive disease in people undergoing haematopoietic stem cell transplantation.The Cochrane database of systematic reviews · 2015Pooled it
- Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure.Blood · 2016Trial
- Defibrotide for the treatment of severe hepatic veno-occlusive disease and multiorgan failure after stem cell transplantation: a multicenter, randomized, dose-finding trial.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2010Trial
- Osteopetrorickets and calcium homeostasis in children with osteopetrosis: an endocrinological single-center study.Frontiers in endocrinology · 2026Article
- Platelet aggregation elicits FasL expression and hepatocyte apoptosis in sinusoidal obstruction syndrome.Scientific reports · 2025Article
- Comparison of busulfan pharmacokinetics between four-times-daily and once-daily administration in pediatric patients: a preliminary prospective observational trial.International journal of hematology · 2025Observational
- [Clinical analysis of allogeneic hematopoietic cell transplantation in 9 patients with hematological malignancies complicated by Gilbert's syndrome].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2024Article
- Case report: Gene mutations and clinical characteristics of four patients with osteopetrosis.Frontiers in pediatrics · 2023Article
- Article
- [Chinese expert consensus on the diagnosis and management of sinusoidal obstruction syndrome after hematopoietic stem cell transplantation (2022)].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2022Article
- Hepatic sinusoidal obstruction syndrome/veno-occlusive disease after hematopoietic cell transplantation: historical and current considerations in Korea.The Korean journal of internal medicine · 2021Review
- Autosomal recessive osteopetrosis: mechanisms and treatments.Disease models & mechanisms · 2021Review
- Improved detection of sinusoidal obstructive syndrome using pediatric-AYA diagnostic criteria and severity grading.Bone marrow transplantation · 2021Article
- Incidence of Anicteric Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome and Outcomes with Defibrotide following Hematopoietic Cell Transplantation in Adult and Pediatric Patients.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2020Article
- Diagnosis and Treatment of VOD/SOS After Allogeneic Hematopoietic Stem Cell Transplantation.Frontiers in immunology · 2020Review
- Systematic review of defibrotide studies in the treatment of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS).Bone marrow transplantation · 2019Article
- Assessment of the proposed EBMT pediatric criteria for diagnosis and severity grading of sinusoidal obstruction syndrome.Bone marrow transplantation · 2019Article
- Stem cell transplantation for osteopetrosis in patients beyond the age of 5 years.Blood advances · 2019Article
- Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy.Bone marrow transplantation · 2018Article
Corrections and comments
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Authors and funding
7 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Malignant infantile osteopetrosis (MIOP) is a rare hereditary disorder of osteoclast function, which can be reversed by hematopoietic stem cell transplantation (SCT). We observed a high incidence of hepatic veno-occlusive disease (VOD) in transplanted patients and explored the prevention of this complication by using defibrotide (DF) as a prophylaxis. Twenty children with MIOP were consecutively transplanted in our center between 1996 and 2005. Eleven of these patients were transplanted between 1996 and 2001 and experienced an overall incidence of VOD of 63.6% (7/11). VOD was severe in three patients and one patient succumbed to VOD-related multi-organ failure. Owing to this very high incidence of VOD, DF prophylaxis was initiated in nine patients consecutively transplanted between 2001 and 2005. In this group, only one patient (11.1%) was diagnosed with moderate VOD. We report here a very high risk in patients with MIOP to develop VOD after transplantation. Prophylactic DF was implemented in our current transplant protocol and reduced the VOD rate significantly in this high-risk population.
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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.