Prognostic factors for survival and response after high‐dose therapy and autologous stem cell transplantation in systemic AL amyloidosis: a report on 21 patients

• Published in: British journal of haematology 1998-06, Vol.101 (4), p.766-769
• Main Authors: Moreau, Philippe, Leblond, Véronique, Bourquelot, Priscille, Facon, Thierry, Huynh, Anne, Caillot, Denis, Hermine, Olivier, Attal, Michel, Hamidou, Mohamed, Nedellec, Gérard, Ferrant, Augustin, Audhuy, Bruno, Bataille, Régis, Milpied, Noël, Harousseau, Jean‐Luc
• Format: Article
• Language: English
• Subjects: Adult; Amyloidosis - radiotherapy; Amyloidosis - therapy; Antineoplastic agents; Antineoplastic Agents, Alkylating - therapeutic use; autologous stem cell transplantation; Biological and medical sciences; Combined Modality Therapy; Combined treatments (chemotherapy of immunotherapy associated with an other treatment); Disease-Free Survival; Female; Hematology; Hematopoietic Stem Cell Mobilization - adverse effects; Hematopoietic Stem Cell Mobilization - methods; Hematopoietic Stem Cell Transplantation - adverse effects; Hematopoietic Stem Cell Transplantation - methods; Humans; Male; Medical sciences; Melphalan - therapeutic use; Middle Aged; Pharmacology. Drug treatments; Prognosis; Retrospective Studies; systemic AL amyloidosis; Transplantation, Autologous; Whole-Body Irradiation
• ISSN: 0007-1048; 1365-2141
• DOI: 10.1046/j.1365-2141.1998.00772.x

We retrospectively investigated the feasibility and the toxicity of autologous stem cell transpantation (ASCT) in 21 cases of systemic amyloidosis (AL). The conditioning regimens consisted of high‐dose melphalan (HDM) alone (n = 18) or in combination with 12 Gy total body irradiation (n = 3). Toxic death rate was high: 9/21 patients (43%) died within the first month following ASCT, and 10/12 surviving patients achieved a response. With a median follow‐up of 14 months, the OS and the EFS rates at 4 years were 57.1% (±10.8) and 29.9% (±14.5) respectively for the whole group. The major prognostic factor for both response and survival was the number of clinical manifestations at the time of ASCT, of the following five criteria, i.e. creatinine clearance  3000 mg/24 h, congestive heart failure, neuropathy, or hepatomegaly associated with alkaline phosphatase level > 200 IU/l. For patients presenting with two or more clinical manifestations the 4‐year OS and EFS were both 11.1% compared with 91.7% and 46.3% respectively in patients with fewer than two clinical manifestations at the time of ACST. We conclude that ASCT is feasible in AL in a subset of patients with fewer than two clinical manifestations at the time of ASCT. Given the severe extra‐haematological toxicity, ASCT should not be considered in other cases.