Evidence map›Paper›PMID 42152527›Full record

ArticleBritish journal of haematology2026

Outcomes of an optimized ciclosporin-free haploidentical HSCT protocol in paediatric patients with cerebral adrenoleukodystrophy.

Fanghua Ye, Bin Fu, Keke Chen, Xiaodan Xia, Yurong Li, Leyuan Wang, Hui Yang, Wenjun Deng, Zhongqing Wang, Liangchun Yang

Abstract read
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In one paragraph

Article in British journal of haematology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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0citing papers in PubMed
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1 · What the graph read from it

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.

2 · The registry

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3 · Its place in the literature

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4 · The record

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5 · Who and what money

Authors and funding

10 authors.

Fanghua YeDepartment of Pediatrics, Xiangya Hospital Central South University, Changsha, China.
Bin FuDepartment of Hematology, Xiangya Hospital Central South University, Changsha, China.
Keke ChenDepartment of Pediatrics, Hunan Provincial People's Hospital (The First Affiliated Hospital of Hunan Normal University), Changsha, China.
Xiaodan XiaDepartment of Pediatrics, Xiangya Hospital Central South University, Changsha, China.
Yurong LiDepartment of Pediatrics, Xiangya Hospital Central South University, Changsha, China.
Leyuan WangDepartment of Pediatrics, Xiangya Hospital Central South University, Changsha, China.
Hui YangDepartment of Pediatrics, Xiangya Hospital Central South University, Changsha, China.
Wenjun DengDepartment of Pediatrics, Xiangya Hospital Central South University, Changsha, China.
Zhongqing WangDepartment of Ultrasound Imaging, Xiangya Hospital Central South University, Changsha, China.ORCID https://orcid.org/0000-0002-4636-0162
Liangchun YangDepartment of Pediatrics, Xiangya Hospital Central South University, Changsha, China.ORCID https://orcid.org/0000-0001-6766-6577

Funding

Changsha Science and Technology Project kq2403024National Natural Science Foundation of China 82500228Natural Science Foundation of Hunan Province 2024JJ5598Natural Science Foundation of Hunan Province 2025JJ60564
6 · The paper itself

Abstract

Haploidentical haematopoietic stem cell transplantation (haplo-HSCT) expands donor availability for cerebral adrenoleukodystrophy (cALD). However, conventional graft-versus-host disease (GVHD) prophylaxis based on calcineurin inhibitors like ciclosporin (CsA) poses a potential neurotoxicity risk that may exacerbate neurological injury. We retrospectively analysed 26 cALD patients who underwent haplo-HSCT with an optimized ciclosporin-free GVHD prophylaxis regimen consisting of anti-thymocyte globulin (ATG; 4 mg/kg), post-transplant cyclophosphamide (PTCy) and mycophenolate mofetil. Neutrophil and platelet engraftment each occurred at a median of 15 days (ranges, 13-26 and 9-33 days respectively). The cumulative incidence (CI) of grades II-IV acute GVHD by day 100 was 18.49% ± 8.37%, and the 3-year CI of moderate-to-severe chronic GVHD was 9.32% ± 6.28%. After a median follow-up of 29 months, overall survival was 90.4% ± 6.6% and major functional disability-free survival was 69.0% ± 9.1%. Post-transplantation, plasma C26:0 levels and the C24:0/C22:0 and C26:0/C22:0 ratios were markedly reduced. Magnetic resonance imaging (MRI) at a mean of 7.3 ± 5.9 months post-HSCT showed largely preserved LOES scores without significant progression. Our experience supports the safety and efficacy of this optimized ciclosporin-free haplo-HSCT approach for patients with cALD, warranting further validation in prospective studies.

Indexed as

AdrenoleukodystrophyHematopoietic Stem Cell TransplantationTransplantation, HaploidenticalAdolescentAntilymphocyte SerumChildChild, PreschoolCyclophosphamideCyclosporineFemaleGraft vs Host DiseaseHumansImmunosuppressive AgentsInfantMaleMycophenolic AcidAntilymphocyte SerumCyclophosphamideCyclosporineImmunosuppressive AgentsMycophenolic Acidcerebral adrenoleukodystrophyhaplo‐HSCToutcomespaediatricpost‐transplantation cyclophosphamide

Identifiers

PMID42152527
PMCPMC13461975

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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.