Haploidentical Peripheral Blood Stem Cell Transplantation with Post-Transplantation Cyclophosphamide in Children with Advanced Acute Leukemia with Fludarabine-, Busulfan-, and Melphalan-Based Conditioning

被引:45
作者
Jaiswal, Sarita Rani [1 ,2 ]
Chakrabarti, Aditi [2 ]
Chatterjee, Sumita [2 ,3 ]
Bhargava, Sneh [1 ]
Ray, Kunal [4 ,5 ]
O'Donnell, Paul [6 ]
Chakrabarti, Suparno [1 ,2 ]
机构
[1] Dharamshila Hosp & Res Ctr, Dept Blood & Marrow Transplantat, New Delhi 110096, India
[2] Manashi Chakrabarti Fdn, Kolkata, India
[3] Apollo Gleneagles Hosp, Kolkata, India
[4] Indian Inst Chem Biol, CSIR, Kolkata, India
[5] CSIR CRRI, Acad Sci & Innovat Res, New Delhi, India
[6] Fred Hutchinson Canc Res Ctr, 1124 Columbia St, Seattle, WA 98104 USA
关键词
Haploidentical; Post-transplantation cyclophosphamide; Leukemia; Peripheral blood stem cells; Pediatric; BONE-MARROW-TRANSPLANTATION; HIGH-DOSE BUSULFAN; ACUTE MYELOID-LEUKEMIA; SEVERE APLASTIC-ANEMIA; HEMATOLOGIC MALIGNANCIES; VENOOCCLUSIVE DISEASE; PEDIATRIC-PATIENTS; SIBLING DONOR; REGIMEN;
D O I
10.1016/j.bbmt.2015.11.010
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Post-transplantation cyclophosphamide (PTCY) therapy has made haploidentical transplantation a global reality in adults, but the literature is largely silent on the feasibility of this approach in children. We conducted a prospective study of 20 patients (median age, 12 years; range, 2-20 years) with advanced acute leukemia to evaluate the feasibility of PTCY-based haploidentical peripheral blood stem cell (PBSC) transplantation in children. The conditioning regimen comprised fludarabine, i.v. busulfan, and melphalan (Flu-Bu-Mel). PTCY on days +3 and +4 was followed by mycophenolate mofetil for 14-21 days and cyclosporine for 60 days. Thirteen patients (65%) had refractory or relapsed myelogenous leukemia, and the remainder had high-risk lymphoblastic leukemia. Prompt engraftment was noted at a median of 14 days, with full donor chimerism by day +28. The cumulative incidence of acute and chronic graft-versus-host disease was 35% and 5%, respectively. Nonrelapse mortality at 1 year was 20%. The incidence of disease progression was 25.7%. The actuarial overall survival at 2 years was 64.3% (95% confidence interval, 53.4%-75.2%). Our data suggest that Flu-Bu-Mel based conditioning followed by PTCY-based haploidentical PBSC transplantation with reduced duration of immunosuppression is feasible in pediatric patients with advanced leukemia. (C) 2016 American Society for Blood and Marrow Transplantation.
引用
收藏
页码:499 / 504
页数:6
相关论文
共 34 条
[1]  
[Anonymous], BIOL BLOOD MARROW S2
[2]   Treatment of high-risk acute leukemia with T-cell-depleted stem cells from related donors with one fully mismatched HLA haplotype [J].
Aversa, F ;
Tabilio, A ;
Velardi, A ;
Cunningham, I ;
Terenzi, A ;
Falzetti, F ;
Ruggeri, L ;
Barbabietola, G ;
Aristei, C ;
Latini, P ;
Reisner, Y ;
Martelli, MF .
NEW ENGLAND JOURNAL OF MEDICINE, 1998, 339 (17) :1186-1193
[3]   Prognostic Value of Minimal Residual Disease Quantification Before Allogeneic Stem-Cell Transplantation in Relapsed Childhood Acute Lymphoblastic Leukemia: The ALL-REZ BFM Study Group [J].
Bader, Peter ;
Kreyenberg, Hermann ;
Henze, Guenter H. R. ;
Eckert, Cornelia ;
Reising, Miriam ;
Willasch, Andre ;
Barth, Andrea ;
Borkhardt, Arndt ;
Peters, Christina ;
Handgretinger, Rupert ;
Sykora, Karl-Walter ;
Holter, Wolfgang ;
Kabisch, Hartmut ;
Klingebiel, Thomas ;
von Stackelberg, Arend .
JOURNAL OF CLINICAL ONCOLOGY, 2009, 27 (03) :377-384
[4]   T-cell replete haploidentical donor transplantation using post-transplant CY: an emerging standard-of-care option for patients who lack an HLA-identical sibling donor [J].
Bashey, A. ;
Solomon, S. R. .
BONE MARROW TRANSPLANTATION, 2014, 49 (08) :999-1008
[5]   VENOOCCLUSIVE DISEASE OF THE LIVER - DEVELOPMENT OF A MODEL FOR PREDICTING FATAL OUTCOME AFTER MARROW TRANSPLANTATION [J].
BEARMAN, SI ;
ANDERSON, GL ;
MORI, M ;
HINDS, MS ;
SHULMAN, HM ;
MCDONALD, GB .
JOURNAL OF CLINICAL ONCOLOGY, 1993, 11 (09) :1729-1736
[6]   Individual patient data meta-analysis of allogeneic peripheral blood stem cell transplant vs bone marrow transplant in the management of hematological malignancies: indirect assessment of the effect of day 11 methotrexate administration [J].
Bensinger, W. .
BONE MARROW TRANSPLANTATION, 2006, 38 (08) :539-546
[7]   Bone Marrow Compared with Peripheral Blood Stem Cells for Haploidentical Transplantation with a Nonmyeloablative Conditioning Regimen and Post-transplantation Cyclophosphamide [J].
Castagna, Luca ;
Crocchiolo, Roberto ;
Furst, Sabine ;
Bramanti, Stefania ;
El Cheikh, Jean ;
Sarina, Barbara ;
Granata, Angela ;
Mauro, Elisa ;
Faucher, Catherine ;
Mohty, Bilal ;
Harbi, Samia ;
Chabannon, Christian ;
Carlo-Stella, Carmelo ;
Santoro, Armando ;
Blaise, Didier .
BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2014, 20 (05) :724-729
[8]  
Chakrabarti Suparno, 2007, Curr Stem Cell Res Ther, V2, P163, DOI 10.2174/157488807780599211
[9]   Allogeneic stem cell transplantation for children with acquired severe aplastic anaemia: a retrospective study by the Viva-Asia Blood and Marrow Transplantation Group [J].
Chen, Jing ;
Lee, Vincent ;
Luo, Cheng Juan ;
Chiang, Alan Kwok Shing ;
Hongeng, Suradej ;
Tan, Poh Lin ;
Tan, Ah Moy ;
Sanpakit, Kleebsabai ;
Li, Chun Fu ;
Lee, Anselm Chi-wai ;
Chua, Hsin Chieh ;
Okamoto, Yasuhiro .
BRITISH JOURNAL OF HAEMATOLOGY, 2013, 162 (03) :383-391
[10]   Toxic injury to hepatic sinusoids: Sinusoidal obstruction syndrome (veno-occlusive disease) [J].
DeLeve, LD ;
Shulman, HM ;
McDonald, GB .
SEMINARS IN LIVER DISEASE, 2002, 22 (01) :27-41