Ruxolitinib and methylprednisolone for treatment of patients with relapsed/refractory multiple myeloma

Area of Science:

• Oncology
• Hematology
• Pharmacology

Background:

• Janus kinase (JAK) inhibitors are effective for autoimmune disorders and myeloproliferative neoplasms.
• Their efficacy in other cancers, like multiple myeloma (MM), is not well-established.
• Heavily pre-treated MM patients often require novel therapeutic strategies.

Purpose of the Study:

• To evaluate the efficacy and tolerability of oral ruxolitinib combined with methylprednisolone in patients with progressive multiple myeloma.
• To assess the clinical activity of this combination in patients who have received extensive prior therapies, including proteasome inhibitors and lenalidomide.
• To explore the potential of JAK inhibitors beyond myeloproliferative neoplasms.

Main Methods:

• A clinical study involving 29 heavily pre-treated multiple myeloma patients.
• Patients received oral ruxolitinib (15 mg twice daily) and oral methylprednisolone (40 mg every other day).
• Follow-up was conducted until April 28, 2022, with analysis of cytogenetics and fluorescent in situ hybridization (FISH).

Main Results:

• The overall response rate was 31%, with a median duration of response of 13.1 months.
• Median progression-free survival was 3.4 months.
• The combination therapy was well-tolerated and showed activity even in patients with high-risk cytogenetics.

Conclusions:

• The combination of ruxolitinib and methylprednisolone demonstrates significant clinical activity in heavily pre-treated multiple myeloma patients.
• This study is the first to show the efficacy of JAK inhibitors combined with steroids for MM.
• This finding expands the potential therapeutic applications of JAK inhibitors to cancers beyond myeloproliferative neoplasms.