Blood Res 2013; 48(3):
Published online September 25, 2013
https://doi.org/10.5045/br.2013.48.3.178
© The Korean Society of Hematology
1Department of Internal Medicine, Chungbuk National University Hospital, Cheongju, Korea.
2Department of Internal Medicine, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea.
Correspondence to : Correspondence to Je-Hwan Lee, M.D., Ph.D. Department of Internal Medicine, Asan Medical Center, University of Ulsan College of Medicine, Asanbyeongwon-gil 86, Songpa-gu, Seoul 138-736, Korea. Tel: +82-2-3010-3218, Fax: +82-2-3010-6885, jhlee3@amc.seoul.kr
This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/3.0/) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
In adults, the 2 main types of myelodysplastic/myeloproliferative neoplasms (MDS/MPN) are chronic myelomonocytic leukemia (CMML) and atypical chronic myeloid leukemia (aCML). Both are associated with a poor prognosis. Allogeneic hematopoietic cell transplantation (HCT) is the only known curative treatment modality for these diseases, but data on outcomes following such treatment are limited. We analyzed the outcomes of patients with MDS/MPN after allogeneic HCT.
This retrospective study included 10 patients with MDS/MPN who received allogeneic HCT at Asan Medical Center from 2002 to 2010. Of these 10 patients, 7 had CMML, 2 had aCML, and 1 had unclassifiable MDS/MPN. Five patients received a myeloablative conditioning (MAC) regimen (busulfan-cyclophosphamide), and 5 received reduced-intensity conditioning (RIC) regimen.
Neutrophil engraftment was achieved in all patients. After a median follow-up of 47.5 months among surviving patients, 4 had relapsed and 5 had died. There was only 1 treatment-related death. The 5-year rates of overall, relapse-free, and event-free survival were 42.2%, 51.9%, and 46.7%, respectively. Relapse was the leading cause of treatment failure, and all relapses were observed in patients who had received RIC and who did not develop chronic graft-versus-host disease.
Allogeneic HCT can induce durable remission in patients with MDS/MPN, but RIC cannot replace MAC in patients eligible for myeloablative treatments.
Keywords MDS/MPN, Allogeneic HCT, Reduced-intensity, Relapse
Blood Res 2013; 48(3): 178-184
Published online September 25, 2013 https://doi.org/10.5045/br.2013.48.3.178
Copyright © The Korean Society of Hematology.
Sung-Nam Lim1, Je-Hwan Lee2*, Jung-Hee Lee2, Dae-Young Kim2, Sung Doo Kim2, Young-A Kang2, Young-Shin Lee2, and Kyoo-Hyung Lee2
1Department of Internal Medicine, Chungbuk National University Hospital, Cheongju, Korea.
2Department of Internal Medicine, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea.
Correspondence to: Correspondence to Je-Hwan Lee, M.D., Ph.D. Department of Internal Medicine, Asan Medical Center, University of Ulsan College of Medicine, Asanbyeongwon-gil 86, Songpa-gu, Seoul 138-736, Korea. Tel: +82-2-3010-3218, Fax: +82-2-3010-6885, jhlee3@amc.seoul.kr
This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/3.0/) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
In adults, the 2 main types of myelodysplastic/myeloproliferative neoplasms (MDS/MPN) are chronic myelomonocytic leukemia (CMML) and atypical chronic myeloid leukemia (aCML). Both are associated with a poor prognosis. Allogeneic hematopoietic cell transplantation (HCT) is the only known curative treatment modality for these diseases, but data on outcomes following such treatment are limited. We analyzed the outcomes of patients with MDS/MPN after allogeneic HCT.
This retrospective study included 10 patients with MDS/MPN who received allogeneic HCT at Asan Medical Center from 2002 to 2010. Of these 10 patients, 7 had CMML, 2 had aCML, and 1 had unclassifiable MDS/MPN. Five patients received a myeloablative conditioning (MAC) regimen (busulfan-cyclophosphamide), and 5 received reduced-intensity conditioning (RIC) regimen.
Neutrophil engraftment was achieved in all patients. After a median follow-up of 47.5 months among surviving patients, 4 had relapsed and 5 had died. There was only 1 treatment-related death. The 5-year rates of overall, relapse-free, and event-free survival were 42.2%, 51.9%, and 46.7%, respectively. Relapse was the leading cause of treatment failure, and all relapses were observed in patients who had received RIC and who did not develop chronic graft-versus-host disease.
Allogeneic HCT can induce durable remission in patients with MDS/MPN, but RIC cannot replace MAC in patients eligible for myeloablative treatments.
Keywords: MDS/MPN, Allogeneic HCT, Reduced-intensity, Relapse
Survival differences between myeloablative conditioning and reduced-intensity conditioning;
Table 1 . Patient characteristics at the time of hematopoietic cell transplantation..
Abbreviations: UPN, unique patient number; M, male; F, female; Dx, diagnosis; CMML, chronic myelomonocytic leukemia; aCML, atypical chronic myeloid leukemia; MDS/MPN-U, myelodysplastic syndrome/myeloproliferative neoplasm-unclassifiable; HCT, hematopoietic cell transplantation; Tx, treatment; FLAG-D, fludarabine, cytarabine, granulocyte colony-stimulating factor plus daunorubicin; KPS, Karnofsky performance score; HCT-CI, hematopoietic cell transplantation-comorbidity index; MDAPS, M.D. Anderson Prognostic Score; INT, intermediate; CR, complete remission; BM, bone marrow; NHL, non-Hodgkin lymphoma..
Table 2 . Transplantation data..
a)Number of incompatibilities among 8 ABDR loci between donor and recipient..
Abbreviations: UPN, unique patient number; HLA, human leukocyte antigen; GVHD, graft-vs-host disease; CSA, cyclosporin; MTX, methotrexate; BuCy, busulfan+cytoxan; BuFluATG, busulfan+fludarabine+thymoglobulin; BuFluCampath, busulfan+fludarabine+alemtuzumab; MelFlu, melphalan-fludarabine; BM, bone marrow; PB, peripheral blood stem cell; MNC, mononuclear cell..
Table 3 . Post-transplant outcomes..
Abbreviations: UPN, unique patient number; ANC, absolute neutrophil counts ≥0.5×109/L; PLT, platelet counts ≥20×109/L; Reti, absolute reticulocyte counts ≥1.0%; CC, complete chimerism; MC, mixed chimerism; GVHD, graft-versus-host disease; Gr, grade; NE, not evaluable; SOS, sinusoidal obstruction syndrome; CMV, cytomegalovirus; IP, interstitial pneumonitis; F/U, follow-up; NED, no evidence of disease; HCT, hematopoietic cell transplantation; DLI, donor lymphocyte infusion; AML, acute myeloid leukemia..
Table 4 . Published data for post-transplant outcomes in MDS/MPN (except juvenile myelomonocytic leukemia)..
Abbreviations: MDS/MPN, myelodysplastic syndrome/myeloproliferative neoplasm; MDS/MPN-U, MDS/MPN-unclassified; CMML, chronic myelomonocytic leukemia; aCML, atypical chronic myeloid leukemia; MF, myelofibrosis; MRD, matched related donor; MMRD, mismatched related donor; MUD, matched unrelated donor; MMUD, mismatched unrelated donor; BM, bone marrow; PB, peripheral blood; TBI, total body irradiation; MAC, myeloablative conditioning; RIC, reduced-intensity conditioning; BuCy, busulfan+cytoxan; ATG, antithymocyte globulin; FLAMSA, fludarabine+amsacrine+cytarabine; BuFlu, busulfan+fludarabine; MelFlu, melphalan+fludarabine; FAI, fludarabine+cytarabine+idarubicin; BuFluATG, busulfan+fludarabine+ATG; BuFluCampath, busulfan+fludarabine+alemtuzumab; TRM, treatment-related mortality; OS, overall survival; RFS, relapse-free survival; DFS, disease-free survival; EFS, event-free survival..
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Survival differences between myeloablative conditioning and reduced-intensity conditioning;