Related Experiment Video
Updated: Jun 6, 2026
Percutaneous Hepatic Perfusion (PHP) with Melphalan as a Treatment for Unresectable Metastases Confined to the Liver
Published on: July 31, 2016
Oral cyclic melphalan and dexamethasone for patients with AL amyloidosis
Vaishali Sanchorawala1, David C Seldin, John L Berk
1Section of Hematology-Oncology, Department of Medicine, Boston University School of Medicine, 820 Harrison Avenue, Boston, MA 02118, USA. vaishali.sanchorawala@bmc.org
Purpose:
Aggressive treatment of amyloid light chain (AL) amyloidosis with high-dose intravenous melphalan followed by autologous stem cell transplantation (HDM/SCT) is effective in inducing hematologic remission and clinical improvement. However, only selected patients with AL amyloidosis are eligible for HDM/SCT because of amyloid-associated organ dysfunction.
Patients And Methods:
We report on 70 patients with AL amyloidosis treated with oral cyclic melphalan and dexamethasone.
Results:
Of 48 evaluable patients who survived and returned for follow-up assessment, 6 patients (13%) achieved a complete hematologic response and 12 patients (25%) a partial hematologic response. Responses were non-inferior for patients receiving weekly "low-dose" dexamethasone compared with those receiving 4 day pulses. Median survival for the 70 patients has not yet been reached with a median follow-up of 17 months. Nineteen patients (27%) received additional treatment leading to improvement in survival.
Conclusion:
Melphalan/dexamethasone can lead to hematologic responses and improvement in survival, particularly for those who can receive additional treatment for AL amyloidosis.
