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Translational Medicine / 转化医学Pediatric Acute Lymphoblastic Leukemia

Ching-Hon Pui

裴正宏

MD

🏢St. Jude Children's Research Hospital(圣裘德儿童研究医院)🌐USA

Department Chair Emeritus, Oncology; Fahad Nassar Al-Rashid Chair of Leukemia Research; Member, St. Jude Children's Research Hospital肿瘤学系荣誉主任,白血病研究教授

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Key Papers
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Awards
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Key Contributions

👥Biography 个人简介

Ching-Hon Pui, MD is one of the world's foremost authorities on pediatric acute lymphoblastic leukemia (ALL), having spent over four decades at St. Jude Children's Research Hospital transforming it from a uniformly fatal disease to one with cure rates exceeding 90% in children. He pioneered risk-stratified, total-therapy approaches—collectively known as the St. Jude Total Therapy protocols—that systematically replaced cranial irradiation with intrathecal chemotherapy for CNS prophylaxis, dramatically reducing long-term neurocognitive toxicity while maintaining disease control. His work on minimal residual disease (MRD) monitoring established a new paradigm for treatment response assessment and early intensification decisions. Dr. Pui has authored or co-authored more than 1,000 peer-reviewed publications and his protocols have become the global benchmark for pediatric ALL management.

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🧪Research Fields 研究领域

Pediatric Acute Lymphoblastic Leukemia (ALL)儿童急性淋巴细胞白血病
Risk-Adapted Chemotherapy风险分层化疗
CNS-Directed Therapy中枢神经系统定向治疗
Minimal Residual Disease微小残留病变检测
Leukemia Genomics白血病基因组学

🎓Key Contributions 主要贡献

St. Jude Total Therapy Protocols for Pediatric ALL

Designed and led successive generations of the St. Jude Total Therapy trials (Total XV, XVI) achieving >90% 5-year event-free survival in childhood ALL, and demonstrating that cranial irradiation could be entirely eliminated without compromising CNS control through optimized intrathecal and systemic chemotherapy intensification.

Elimination of Prophylactic Cranial Irradiation

Conducted landmark trials proving that prophylactic cranial irradiation could be replaced by intrathecal chemotherapy in the vast majority of patients, sparing children from radiation-induced neurocognitive sequelae, growth failure, and secondary brain tumors—a change now adopted worldwide.

MRD-Guided Treatment Stratification

Pioneered the use of end-induction and end-consolidation minimal residual disease (MRD) measurement by flow cytometry and PCR to guide treatment intensification and deescalation, establishing MRD as the strongest independent prognostic factor in childhood ALL.

Genomic Subtype Discovery and Risk Classification

Contributed to the identification of novel ALL genomic subtypes including Philadelphia chromosome-like ALL (Ph-like ALL), enabling targeted therapy with tyrosine kinase inhibitors and reclassifying patients previously misassigned to standard-risk groups.

Representative Works 代表性著作

[1]

Improved outcome for children with acute lymphoblastic leukemia: results of Total Therapy Study XIIIB at St Jude Children's Research Hospital

Blood (2004)

Demonstrated 80% 5-year EFS for childhood ALL using MRD-guided risk stratification and intensified CNS-directed chemotherapy without cranial irradiation.

[2]

Treating childhood acute lymphoblastic leukemia without cranial irradiation

New England Journal of Medicine (2009)

Landmark trial (Total XV) showing that cranial irradiation could be entirely eliminated in childhood ALL achieving 93.7% 5-year EFS, establishing the modern CNS prophylaxis standard.

[3]

Philadelphia chromosome-like acute lymphoblastic leukemia

Nature Reviews Clinical Oncology (2017)

Comprehensive review defining Ph-like ALL as a high-risk genomic subtype with targetable kinase fusions, forming the basis for ongoing clinical trials with TKIs.

[4]

Outcome of treatment in childhood acute lymphoblastic leukaemia with rearrangements of the 11q23 chromosomal region

Lancet (2002)

Defined the poor-prognosis features of MLL-rearranged infant and childhood ALL, guiding intensification strategies for this high-risk subset.

🏆Awards & Recognition 奖项与荣誉

🏆American Society of Hematology (ASH) E. Donnall Thomas Lecture and Prize
🏆American Society of Clinical Oncology (ASCO) Pediatric Oncology Award
🏆International Society of Paediatric Oncology (SIOP) Giulio J. D'Angio Award
🏆St. Baldrick's Foundation Robert J. Arceci Innovation Award

📄Data Sources 数据来源

Last updated: 2026-04-06 | All information from publicly available academic sources

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