Autologous stem cell transplantation in AL amyloidosis: Muddy waters.

ABSTRACT

Immunoglobulin light chain (AL) amyloidosis is a malignant plasma cell dyscrasia causing multi-organ morbidity. High dose melphalan and autologous stem cell transplantation (ASCT) is a preferred consolidation approach and is safe with improved patient selection criteria. With the advent of bortezomib and daratumumab based induction therapy, nearly all patients can achieve deep hematological responses but follow up for daratumumab based induction is short. Consequently, the traditional approach of induction followed by ASCT is called into question. Given the multi-organ involvement of AL, endpoints beyond depth of response and hematological progression free survival (PFS) are important. Major organ dysfunction PFS (MOD-PFS) adds to PFS and is a composite endpoint of PFS, renal and cardiac organ progression, and overall survival. It is currently unknown which consolidative approach (ASCT or non-ASCT) will generate improved outcomes across the MOD-PFS spectrum a question the recently opened S2213 trial will attempt to answer.