Decision analysis of allogeneic bone marrow transplantation versus immunosuppressive therapy for young adult patients with aplastic anemia

• Published in: International journal of hematology Vol. 117; no. 5; pp. 660 - 668
• Main Authors: Kanda, Yoshinobu, Usuki, Kensuke, Inagaki, Mitsuhiro, Ohta, Akiko, Ogasawara, Yoji, Obara, Naoshi, Kako, Shinichi, Kurokawa, Mineo, Shimada, Naoki, Suzuki, Takahiro, Hama, Asahito, Yamaguchi, Hiroki, Nakao, Shinji, Yamazaki, Hirohito
• Format: Journal Article
• Language: English
• Published: Singapore Springer Nature Singapore 01.05.2023; Springer Nature B.V
• Subjects: Allografts; Anemia; Anemia, Aplastic - drug therapy; Aplastic anemia; Availability; Bone marrow; Bone marrow transplantation; Bone Marrow Transplantation - adverse effects; Cyclosporins; Data analysis; Decision analysis; Decision Support Techniques; Globulins; Graft vs Host Disease - drug therapy; Graft vs Host Disease - etiology; Graft vs Host Disease - prevention & control; Graft-versus-host reaction; Hematology; Hematopoietic Stem Cell Transplantation; Histocompatibility antigen HLA; Humans; Immunosuppression Therapy - adverse effects; Immunosuppressive agents; Immunosuppressive Agents - therapeutic use; Markov chains; Medicine; Medicine & Public Health; Oncology; Original Article; Patients; Prospective Studies; Response rates; Sensitivity analysis; Siblings; Stem cell transplantation; Thymocytes; Transition probabilities; Transplantation; Young Adult; Young adults; Bone marrow transplantation; Severe aplastic anemia; Immunosuppressive therapy; Decision analysis
• ISSN: 0925-5710; 1865-3774
• DOI: 10.1007/s12185-022-03530-6

Background Allogeneic bone marrow transplantation (BMT) from an HLA-matched sibling donor is recommended as an initial treatment for young patients. However, immunosuppressive therapy (IST) with cyclosporine and anti-thymocyte globulin may be a viable option even when an HLA-identical sibling donor is available. Methods We constructed a Markov model to simulate the 10-year clinical course of patients aged 21–40 years with newly diagnosed severe aplastic anemia. Immediate BMT and IST were compared as an initial treatment assuming the availability of an HLA-identical sibling donor. Transition probabilities after treatment were determined based on a registry data analysis for BMT and a long-term prospective study for IST. Results Quality-adjusted life years (QALYs) after treatment selection were 6.77 for BMT and 6.74 for IST. One-way sensitivity analysis revealed that the utility for being alive without GVHD after BMT, that for being alive with partial response after IST, and the response rate after initial IST strongly affected the results. Conclusions BMT and IST produced similar QALY for young patients with severe aplastic anemia. An estimation of the response rate to the initial IST may enable an individualized comparison between BMT and IST.