Brazilian Consensus Guidelines for Hematopoietic Stem Cell Transplantation - Inborn errors of metabolismo

Gomes, Alessandra Araújo; Rodrigues, Adriana Mello; Fernandes, Juliana Folloni; Daudt, Liane Esteves; Bonfim, Carmem

doi:10.46765/2675-374x.2021v2n4p126

Cited by 1 publication

(2 citation statements)

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“…Currently, our group is following recommendations for post-transplantation patient monitoring as proposed by international guidelines for IEM HSCT. 6,9,25,26 Excellent results are achieved when HSCT is performed during the early stages of these diseases with well-matched donors. 9,14,18 However, due to the high miscegenation of the Brazilian population, fully MUD are typically unavailable.…”

Section: Discussionmentioning

confidence: 99%

“…35 The PDWP-SBTMO strongly recommend the use of MAC regimens with BU, guided by pharmacokinetics or weight-based criteria. 25 GVHD is a severe complication in HSCT for non-malignant diseases and it is associated with elevated mortality rates. 23 In this cohort, there was no observed association between the MAC regimen and the incidence of acute GVHD(aGVHD) in 28 out of 105 patients.…”

Section: Discussionmentioning

confidence: 99%

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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

Rodrigues,

Fernandes,

Gregianin

et al. 2024

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Hematopoietic stem cell transplantation (HSCT) is an established treatment for selected patients with inborn errors of metabolism. In this first report from the PDWP-SBTMO, we included 105 patients transplanted between 1988 and 2021 across six Brazilian HSCT centers. The most prevalent diseases were X-linked adrenoleukodystrophy(n=61) and mucopolysaccharidosis (type I n=20; type II n=10), with a median age at HSCT of 8.7years and 2.1years, respectively. Most conditioning regimens were myeloablative and busulfan-based. With a median follow-up of 6.7years, the 5-years overall survival (OS) was 75% (95% CI, 0.65 - 0.82) with a superior 5-year OS for those transplanted after 2010(87% vs. 63%, p=0.01). Higher risk of death was associated to the use of haploidentical donor (HR8.86, p 0.021), unrelated cord blood (HR 8.76, p 0.005), unrelated donor (HR 5.91, p 0.02) and for HSCT performed before 2010(HR 4.16, p=0.0015). The CI of acute-GVHD was 24.8%, while chronic-GVHD was 9.5%. Major causes of death were infections(n=8), GVHD(n=6) and neurologic progression(n=3). Despite improvements in transplant outcomes since 2011, challenges persist, emphasizing the need for early diagnosis, timely transplantation, and expanding HSCT centers with expertise in the field.

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