Dao-Pei Lu May 2010, Shanghai Hematopoietic Stem Cell Transplantation (HSCT) in China (2010)

1 CSBMT and Syn-HSCT

Establishment of CSBMT (Chinese Society of BMT) Hangzhou, establishment of Chinese BMT Cooperation Group in APBMT and China BMT Group meeting Beijing, establishment of CSBMT in ISH-APD & APBMT meeting Hong-Kong, Chinese BMT Forum

1 st CSBMT Conference (2009, Hong Kong)

CSBMT Newsletter

Syn-HSCT in Chinese --A report of 94 cases from 32 centers in CSBMT

Patient characteristics CSBMT AA AML large granular lymphocytosis Neuroblastoma lymphoma MDS CML ALL n=94

OS of Syn-HSCT (n=94)

DFS of Syn-HSCT (n=94)

Follow-up the longest survivor of HSCT in the world

2 Overview of HSCT in China

HSCT Number in Different Cities in China

HSCT Number in Different Units in China

HSCT indications in China （ 2009 ） MM Other leukemia Autoimmune disease Solid tumor AA

Types of HSCT in China （ 2009 ） Identical sibling Haplo Unrelated Auto Syngeneic

GIAC Principle Combined BM & PB C:C: ATG A:A: Immuno-suppression intensified and prolonged I : G-CSF mobilization GG:GG:

Haploidentical HSCT (Haplo-HSCT) Lu et al. Blood 2006; 107: 3065

Haploidentical Blood and Marrow Transplantation in Hematological Malignancies: A Single- Center Report of 290 patients Tong Wu, Dao-Pei Lu, et al. Beijing Daopei Hospital, Beijing, China August th APBMT

Outstanding Oral Presentation Award (Haplo-HSCT) 14th APBMT (August 2009, Korea)

II-IV aGVHD III-IV aGVHD Days after BMT Percent 34.9% 16.0% Acute GVHD of Haplo-HSCT (Beijing Daopei Hospital, n=290)

standard risk high risk advanced Years after BMT LFS 73.9% 51.0% 22.2% P< LFS of Haplo-HSCT (Beijing Daopei Hospital, n=290)

standard risk high risk advanced Years after BMT OS 77.3% 58.1% 29.5% P< OS of Haplo-HSCT (Beijing Daopei Hospital, n=290)

Indications of Unrelated-HSCT (U-HSCT) （ Beijing Daopei Hospital, n=182)

Acute GVHD of U-HSCT (Beijing Daopei Hospital, n=182)

5yr-OS of U-HSCT (Beijing Daopei Hospital, n=182) 72.2%

5yr-DFS of U-HSCT (Beijing Daopei Hospital, n=182) 64.9%

U-HSCT with Haplo-BM (Beijing Daopei Hospital, n=182)

Successful Rescue of Refractory/Recurrent Myelogenous Leukemia by Allogeneic HSCT and Prophylactic Immunotherapy Beijing & Shanghai Daopei Hospitals

Refractory/recurrent myelogenous leukemia by HSCT and immunotherapy 30 cases (AML 29 ， CML-BC myeloid 1) Median blasts in BM 36 ( )% Types of HSCT: –Matched sibling5 –Unrelated7 –Haploidentical18 Median follow-up 17 (5 － 37) months

Conditioning <40% blasts in BM –Ara-C + BuCy13 >40% blasts in BM –Chemotherapy + reduced BuCy 6 Impaired organs ： –Fludarabine substituted for Cy11

Prophylaxis of Relapse –Immunosuppressants were tapered off early post-HSCT –Prophylactic immunotherapy if no GVHD Cellular: DLI, DC-CIK, NK cells Humoral: IL-2, IFN-a, thymosin

Refractory/recurrent myelogenous leukemia by HSCT and immunotherapy OS EFS

3 Further application of HSCT

A Special Neonatal Case of SCID cured by U-CBT Dao-pei Hospital & Pediatric Hospital, Fu Dan University

Reconstitution PLT WBC N# L#

U-CBT 前

U-CBT 后 5 个月

Familial Hemophagocytic Lymphohistiocytosis (FHL) treated by U-HSCT Beijing Daopei Hospital

Patient Zhang x. 13 yr girl “Lymphoma” was diagnosed and treated in other hospitals. FHL was diagnosed on admission.

Zhang’s Family Tree Diagnosed as FHL-2

MACPF 结构域 野生型 PRF1-168SMACPF 结构域 突变型 PRF1-168N MHD2 结构域 野生型 UNC13D-863GMHD2 结构域 突变型 UNC13D-863D Protein conformation of PRF1 gene mutations Paternal Maternal

Perforin normalization post HSCT of Zhang Y Month

Autologous cord blood transplantation (Auto- CBT) in children Beijing Children’s Hospital (BCH) Beijing Daopei Hospital (BDH)

Auto-CBT in Children Date of HSCT HospitalDiagnosisFollow- up (month) Outcome BCHNeuroblas toma 2CR BDHSAA4CR BDHSAA3CR

Auto-CBT for pediatric SAA Beijing Daopei Hospital Before HSCT

Summary 1 、 CSBMT must play an important role in HSCT continuously. 2 、 Large series of syn-HSCT show excellent outcome in both nonmalignant and malignant hematological diseases.

Summary 3 、 HSCT in China has been developing rapidly. The outcomes of Haplo-HSCT and U-HSCT have achieved top level in the world in experienced units. 4 、 DFS in refractory/recurrent myeloid leukemia treated by allo-HSCT and immunotherapy has been improved significantly.

Summary 5 、 More and more diseases could be treated by HSCT, such as allo- HSCT for inherited diseases (SCID, FHL) and auto-CBT for solid tumor and SAA.

Acknowledgment