Decision analysis of allogeneic bone marrow transplantation versus immunosuppressive therapy for young adult patients with aplastic anemia
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...
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Published in | International journal of hematology Vol. 117; no. 5; pp. 660 - 668 |
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Main Authors | , , , , , , , , , , , , , |
Format | Journal Article |
Language | English |
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Springer Nature Singapore
01.05.2023
Springer Nature B.V |
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Abstract | 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. |
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AbstractList | 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.
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.
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.
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. BackgroundAllogeneic 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.MethodsWe 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.ResultsQuality-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.ConclusionsBMT 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. 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. |
Author | Shimada, Naoki Usuki, Kensuke Nakao, Shinji Hama, Asahito Kurokawa, Mineo Suzuki, Takahiro Kako, Shinichi Yamazaki, Hirohito Obara, Naoshi Yamaguchi, Hiroki Ohta, Akiko Kanda, Yoshinobu Ogasawara, Yoji Inagaki, Mitsuhiro |
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Keywords | Bone marrow transplantation Severe aplastic anemia Immunosuppressive therapy Decision analysis |
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Allogeneic bone marrow transplantation (BMT) from an HLA-matched sibling donor is recommended as an initial treatment for young patients. However,... Allogeneic bone marrow transplantation (BMT) from an HLA-matched sibling donor is recommended as an initial treatment for young patients. However,... BackgroundAllogeneic bone marrow transplantation (BMT) from an HLA-matched sibling donor is recommended as an initial treatment for young patients. However,... BACKGROUNDAllogeneic bone marrow transplantation (BMT) from an HLA-matched sibling donor is recommended as an initial treatment for young patients. However,... |
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SubjectTerms | 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 |
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Title | Decision analysis of allogeneic bone marrow transplantation versus immunosuppressive therapy for young adult patients with aplastic anemia |
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