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Kulagin et al. (Abstract)

Cellular Therapy and Transplantation (CTT), Vol. 3, No. 9
doi: 10.3205/ctt-2010-No9-abstract74
© 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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Bone marrow transplantation and immunosuppressive therapy for acquired aplastic anemia: A 15-year two-center experience

Alexander D. Kulagin1,2, Abdulbasir A. Ganapiev1, Irina K. Golubovskaya1, Irina V. Kruchkova2, Natalia V. Stancheva1, Sergey N. Bondarenko1, Vladimir N. Vavilov1, Igor A. Lisukov1,2, Boris V. Afanasyev1

1R.M. Gorbacheva Memorial Institute of Children Hematology and Transplantation, Pavlov State Medical University, Saint-Petersburg, Russia; 2Institute of Clinical Immunology SB RAMS, Novosibirsk, Russia

Correspondence: Alexander D. Kulagin, R.M. Gorbacheva Memorial Institute of Children Hematology and Transplantation, Saint-Petersburg Pavlov State Medical University, 6/8, Tolstoy str., Saint-Petersburg, 197022, Russia, E-mail: kulagingem@rambler.ru

Abstract

Aim: To evaluate allogeneic bone marrow transplantation (BMT), immunosuppressive treatment (IST) efficacy and long-term outcomes in a large cohort of acquired aplastic anemia (AA) patients.

Methods: We retrospectively analyzed the outcomes of 161 patients (92 M and 69 F, median age 19.5 years, range 1–68) with moderate (MAA 46), severe (SAA 77), and very severe (VSAA 38) AA treated in two centers between 1995 and 2010. The first line therapies were IST in 153 patients (ATG x 1–2 courses and CsA ± daclizumab: 122, CsA alone: 31) and BMT from MRD with Cy + ATG conditioning in 8 patients. In addition, 21 patients were transplanted after fludarabine-based conditioning from MRD (10) or an alternative donor (11) after failed IST (17) and for MDS/AML evolution (4).

Results: There were 8 early (<6 mo) and 17 late (>6 mo) deaths. The median follow-up for living patients was 44 months. The best results were achieved after first line BMT from MRD with a 9-year overall survival (OS) of 100%. Second line BMT from MRD and alternative donors provided an 80% and 44% OS respectively. The main problems of second line BMT were rejection and poor graft function, probably associated with a long-term period of transfusion dependence before transplant (median 38 mo). Extensive chronic GVHD developed in 2 patients who received peripheral blood stem cells.

In the IST group the hematological response was evaluated in 137 patients according to the response criteria of B. Camitta (2000). Forty-three (31%) and 56 (41%) patients achieved CR and PR, respectively. The quality of response was highest in the VSAA group (CR/PR=17/3) compared with the SAA (21/24, p=0.057) and MAA (5/29, p<0.001) groups. Nineteen patients (19%) relapsed and 15 responded again after retreatment with ATG and CsA. Late events included MDS/AML (7, including 5 with monosomy 7, all cases evolved from MAA), trisomy 6 (1), rectal cancer (1) and hemolytic PNH (9). The 10-year OS was 77% without significant difference between the MAA (80%), SAA (80 %), and VSAA (75 %) patients. There was a marked trend towards a better 10-year failure-free survival in very severe disease (37%, 34% and 59% in MAA, SAA, and VSAA respectively).

Conclusions: BMT from MRD and intensive IST provides a high long-term survival rate despite the severity of the disease with the best chance for an actual cure after early BMT. Alternative donor BMT can be a salvage option for patients who fail IST. Long-term results of IST show a rather high incidence of relapse and late events, reflecting the remission state and biological diversity of AA. Early patient referral to an AA expertise center should be carried out because both BMT and IST results worsen if treatment is delayed.

Keywords: aplastic anemia, bone marrow transplantation, immunosuppressive treatment

 

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