What question did this study set out to answer?

To evaluate the efficacy and safety of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in transfusion-dependent pyruvate kinase deficiency (PKD) in Chinese children.

December 8, 2025Blood

Allogeneic hematopoietic stem cell transplantation for transfusion-dependent pyruvate kinase deficiency in Chinese children: A multicenter retrospective study

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Authors

RLRong LiuJiangxi Provincial Cancer Hospital XWXiaodong WangChengdu Medical College XZXiaofan ZhuUniversity of Science and Technology of China

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Implication

Retrospective analysis of allo-HSCT shows 97.1% overall survival in transfusion-dependent pyruvate kinase deficiency patients, highlighting effective treatment options.

Key Points

To evaluate the efficacy and safety of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in transfusion-dependent pyruvate kinase deficiency (PKD) in Chinese children.
Multicenter retrospective study of 35 transfusion-dependent PKD children undergoing allo-HSCT across 13 hospitals.
Data analysis included demographic characteristics, genetic profiles, transplant protocols, and clinical outcomes.
Graft-versus-host disease (GVHD)-free, relapse-free survival (GRFS) defined as the composite endpoint.
Overall survival (OS) and GRFS estimated using Kaplan-Meier methodology.
5-year overall survival (OS) was 97.1%.
85.7% of patients achieved graft-versus-host disease-free, relapse-free survival (GRFS).
Complications included grade 2-4 acute GVHD in 31.4% and chronic GVHD in 22.9%.
One patient died of hepatic veno-occlusive disease (VOD), while others remained disease-free.

Cite This Study

Liu et al. (2025) studied this question.

synapsesocial.com/papers/69362f7f4fa91c937236e528 https://doi.org/10.1182/blood-2025-2521

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Implication

Retrospective analysis of allo-HSCT shows 97.1% overall survival in transfusion-dependent pyruvate kinase deficiency patients, highlighting effective treatment options.

Allogeneic hematopoietic stem cell transplantation for transfusion-dependent pyruvate kinase deficiency in Chinese children: A multicenter retrospective study

Discussion

Member takes

Implication

Key Points

Cite This Study

Allogeneic hematopoietic stem cell transplantation for transfusion-dependent pyruvate kinase deficiency in Chinese children: A multicenter retrospective study

Discussion

Member takes

Implication

Key Points

Cite This Study

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