Allogeneic hematopoietic stem cell transplantation in patients with childhood cerebral adrenoleukodystrophy: A single‐center experience 'Better prognosis in earlier stage'
| Autor: | Hayriye Daloğlu, Akif Yesilipek, Vedat Uygun, Gulsun Karasu, Dayanat Pasayev, Suleimen Zhumatayev, Koray Yalcin, Volkan Hazar, Mustafa Kemal Demir, Seda Öztürkmen, Suna Celen |
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| Rok vydání: | 2021 |
| Předmět: |
Male
medicine.medical_specialty medicine.medical_treatment 030232 urology & nephrology Hematopoietic stem cell transplantation Disease 030230 surgery Single Center 03 medical and health sciences 0302 clinical medicine Internal medicine Adrenal insufficiency medicine Humans Transplantation Homologous Stage (cooking) Adrenoleukodystrophy Child Retrospective Studies Transplantation business.industry Metabolic disorder Age Factors Hematopoietic Stem Cell Transplantation Prognosis medicine.disease Survival Analysis Child Preschool Pediatrics Perinatology and Child Health Cohort business Follow-Up Studies |
| Zdroj: | Pediatric Transplantation. 25 |
| ISSN: | 1399-3046 1397-3142 |
| Popis: | BACKGROUND ALD is a rare X-linked peroxisomal metabolic disorder with many distinct phenotypes of disease that emerge on a wide scale from adrenal insufficiency to fatal cALD which progresses to a vegetative state within a few years. Currently, HSCT is the only treatment method known to stabilize disease progression in patients with cALD. In this study, we aim to report our HSCT experience in patients with cALD and the factors that determine the success of HSCT, as a single-center experience. METHODS The study cohort involves 23 boys with cALD and three patients with ALD trait and new-onset abnormal behavior who underwent allogeneic HSCT between January 2012 and September 2019 in our transplantation center. Loes scoring, NFS, scale and MFD were performed for evaluating the severity of the cerebral disease. The study cohort was divided into two groups according to baseline NFS and Loes score: early-stage (NFS ≤ 1 and Loes score 1 or Loes score ≥9). RESULTS The pretransplant stage of disease impacted both OS and MFD-free survival. The estimated OS and MFD-free survival at 3 years in patients with advanced disease were 46.1% (95% CI 19.0-73.2) and 23.1% (95% CI 0.2-46.0), respectively, and all patients with the early disease were alive (p: .004) and MFD-free (p |
| Databáze: | OpenAIRE |
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