Among patients with multiple myeloma receiving high-dose melphalan conditioning prior to autologous stem cell transplantation (ASCT), oral cryotherapy was the most consistently effective intervention for reducing severe gastrointestinal (GI) mucositis, according to a systematic review presented at the Fourteenth Annual Meeting of the Society of Hematologic Oncology in Houston, Texas.
Amifostine and uproleselan also showed “meaningful promise” for reducing mucositis and delaying severe diarrhea and improving GI quality of life, respectively, but require prospective validation, wrote the study’s authors, led by Vraj Rangrej, MD, of Mayo Clinic in Jacksonville, Florida.
The review included 14 studies with a total of 1,750 patients undergoing high-dose melphalan–conditioned ASCT. In a representative subcohort of 100 patients, 97% had diarrhea (including 40% with grade 3), 88% had nausea, and 60% had vomiting. Patients with grade 2 or higher diarrhea had longer hospitalization duration and increased supportive care needs, and 13% had treatable stool pathogens. Female sex (OR, 3.7) and creatine clearance below 60 mL/min independently predicted severe diarrhea.
The area under the concentration-time curve (AUC) for melphalan varied across patients from 7.6 to 26.6 mg·h/L, and an AUC of 17.5 mg·h/L or higher predicted severe mucositis. In addition, SLC7A5 rs4240803 polymorphism was independently associated with GI toxicity requiring total parenteral nutrition.
For mucositis interventions, oral cryotherapy decreased the rate of grade 3 oral mucositis from 16% to 2% (P=.004). Amifostine decreased the rate of grade 3 or 4 mucositis from 53% to 10% (P=.023), while opioid use decreased the rate from 69% to 20% (P=.005). Palifermin did not show a significant benefit over placebo in chemotherapy-only high-dose melphalan conditioning, regardless of dosing regimen.
Beyond characterizing effective mucositis interventions, the study’s data on AUC variability, SLC7A5 polymorphism, and stool pathogens support individualized melphalan dosing and routine stool testing in patients with multiple myeloma undergoing ASCT, Dr. Rangrej and colleagues concluded.
Reference
Rangrej V, Tripathi S, Kothari V, et al. Novel interventions for gastrointestinal mucositis in multiple myeloma patients receiving high-dose melphalan for autologous stem cell transplantation: a systematic review. Abstract MM-1169. Presented at the 2026 Society of Hematologic Oncology Annual Meeting; September 9-12, 2026; Houston.
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