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Dolgopolov et al. (Abstract)

Cellular Therapy and Transplantation (CTT), Vol. 3, No. 9
doi: 10.3205/ctt-2010-No9-abstract23
© The Authors. This abstract is provided under the following license: Creative Commons Attribution 3.0 Unported

Abstract accepted for "4th Raisa Gorbacheva Memorial Meeting on Hematopoietic Stem Cell Transplantation",
Saint Petersburg, Russia, September 18–20, 2010

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Haploidentical stem cell transplantation in children with advanced malignancies

Igor S. Dolgopolov, Natalia N. Subbotina, Roman I. Pimenov, Vasiliy K. Boyarshinov, George L. Mentkevich

Institute of Pediatric Oncology and Hematology, Russian Cancer Research Center of Russian Academy of Medical Science, Moscow, Russia

Correspondence: Igor S. Dolgopolov, Institute of Pediatric Oncology and Hematology, 24, Kashirskoye sh., 115478, Moscow, Russia, E-mail: irdolg@spam is badrambler.ru

Abstract

Haploidentical SCT is the therapeutic modality for patients lacking an HLA-identical donor. RIC ensures good tolerability and safety, and early stable full donor chimerism with a potential GvT effect. Since 2001 we have performed 44 haplo-transplantations from relatives in 38 pts with poor prognostic malignancies: 9 AML, 4 ALL, 4 CML, 5 JMML, 1 MDS, 3 NHL, 7 NBIV, 4 EWS, and 1 melanoma. The RIC regimen included Fludarabine 180 mg/m2, and ATG 40 mg/kg in combination with Busulfan 8 mg/kg (n=31) or Treosulfan 30000 mg/m2 (n=7). The PBSC with a median number of 6.3 (2–12.5) х106 CD34+cells/kg were infused after incubation in vitro with vincristine and methylprednisolone. T-depletion was not performed. GvHD prophylaxis consisted of short methotrexate and cyclosporine A or tacrolimus.

Three pts with leukemia progression at the time of SCT didn’t recover and died. Four pts with JMML/MDS rejected and relapsed in the first 2 mos after SCT. Thirty-three pts (82.5%) recovered with full donor chimerism. The incidence of acute GvHD was gr. I–II, 22 pts (64%), gr III, 6 pts (18%), and gr. IV, 0%. Acute GvHD was successfully treated with steroids and ATG. The incidence of chronic GvHD was 52% (mainly extensive). Five pts were regrafted (4 JMML/MDS, 1 CML). Two of them engrafted, and one of them is alive and disease-free at 3 years. Nine pts are alive and well with a median follow-up of 41 (8.1–88.5) mos, 20 pts died from relapse, 2 pts died from acute GvHD, and 7 pts from chronic GvHD and infections. The relapse rate at 1 yr was 75% and 33% for solid tumor and leukemia/lymphoma pts. DFS and EFS were 54% and 22% with a median follow-up of 45 and 25 mos for hematological malignancies vs. 20% and 8% at 10 and 8 mos in pts with solid tumors, respectively.

Haploidentical transplantation following reduced-intensive conditioning is feasible and safe if an appropriate GvHD prophylaxis is given. This strategy is applicable to very high-risk and potentially incurable patients if no conventional donor is available.

Keywords: haploidentical transplantation, chronic GvHD, acute GvHD, RIC

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