Abstract
We designed a trial using two sequential cycles of modified high-dose melphalan at 100 mg/m2 and autologous SCT (mHDM/SCT) in AL amyloidosis (light-chain amyloidosis, AL), AL with myeloma (ALM) and host-based high-risk myeloma (hM) patients through SWOG-0115. The primary objective was to evaluate OS. From 2004 to 2010, 93 eligible patients were enrolled at 17 centers in the United States (59 with AL, 9 with ALM and 25 with hM). The median OS for patients with AL and ALM was 68 months and 47 months, respectively, and has not been reached for patients with hM. The median PFS for patients with AL and ALM was 38 months and 16 months, respectively, and has not been reached for patients with hM. The treatment-related mortality (TRM) was 12% (11/93) and was observed only in patients with AL after SCT. Grade 3 and higher non-hematologic adverse events were experienced by 81%, 67% and 57% of patients with AL, ALM and hM, respectively, during the first and second HDM/SCT. This experience demonstrates that with careful selection of patients and use of mHDM for SCT in patients with AL, ALM and hM, even in the setting of a multicenter study, OS can be improved with acceptable TRM and morbidity.
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Acknowledgements
This investigation was supported in part by the following PHS Cooperative Agreement grant numbers awarded by the National Cancer Institute, DHHS: CA32102, CA38926, CA37981, CA76448, CA35431, CA20319, CA76132, CA04919, CA46441, CA14028, CA58861, CA58416, CA12644, CA22433, CA46368, CA45377, CA35090 and CA46113.
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VS received research funding from Celgene, Onyx and Millennium; DCS received research funding from Celgene; and RZO received research funding from Celgene, Onyx, Johnson and Johnson and Millennium. Other authors declare no conflict of interest.
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VS: concept and design, data analysis and interpretation, manuscript writing; AH: concept and design, collection and assembly of data, data analysis and interpretation, final approval of manuscript; DCS: data analysis and interpretation, final approval of manuscript; KTF: collection and assembly of data, data analysis and interpretation, final approval of manuscript; SAF: collection and assembly of data, data analysis and interpretation, final approval of manuscript; RS: concept and design, collection and assembly of data, data analysis and interpretation, concept and design, data analysis and interpretation; BM: final approval of manuscript; HFS: final approval of manuscript; LAH: final approval of manuscript; RMD: final approval of manuscript; ROZ: final approval of manuscript; and BB: concept and design, data analysis and interpretation, final approval of manuscript.
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Sanchorawala, V., Hoering, A., Seldin, D. et al. Modified high-dose melphalan and autologous SCT for AL amyloidosis or high-risk myeloma: analysis of SWOG trial S0115. Bone Marrow Transplant 48, 1537–1542 (2013). https://doi.org/10.1038/bmt.2013.98
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DOI: https://doi.org/10.1038/bmt.2013.98
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