泊马度胺适应症扩大，获批用于卡波西肉瘤

On May 14, 2020, the U.S. Food and Drug Administration (FDA) expanded the indications of pomalidomide to include the treatment of adult patients with acquired immunodeficiency syndrome (AIDS)-related Kaposi's sarcoma after failure of highly active antiretroviral therapy and patients with human immunodeficiency virus (HIV)-negative Kaposi's sarcoma.

Research 12-C-0047

Efficacy was studied in Study 12-C-0047, an open-label, single-arm clinical trial conducted by the National Cancer Institute. Twenty-eight patients (18 HIV-positive, 10 HIV-negative) received 5 mg of pomalidomide orally daily on days 1 through 21 of each 28-day cycle until disease progression or unacceptable toxicity. All HIV-positive patients continued highly active antiretroviral therapy.

The primary efficacy outcome measure was overall response rate, including complete response, clinical complete response and partial response. Investigators assessed response according to the AIDS Clinical Trials Group Oncology Committee Response Criteria for Kaposi's Sarcoma.

Research results

Among 18 HIV-positive patients, the overall response rate was 67% (95% CI = 41%–87%) and the median response duration was 12.5 months (95% CI = 6.5–24.9).

Among 10 HIV-negative patients, the overall response rate was 80% (95% CI = 44%–98%) and the median response time was 10.5 months (95% CI = 3.9–24.2).

The most common adverse reactions in patients receiving pomalidomide include laboratory abnormalities (≥30% of patients) that are decreased absolute neutrophil count or leukocytes, increased creatinine or glucose, rash, constipation, fatigue, decreased hemoglobin, platelets, phosphate, albumin, or calcium, increased ALT, nausea, and diarrhea.

The recommended pomalidomide dose for Kaposi's sarcoma is 5 mg orally once daily with or without food on days 1 through 21 of each 28-day cycle until disease progression or unacceptable toxicity. Prescribing information indicates that patients with AIDS-related Kaposi's sarcoma should continue to use highly active antiretroviral therapy as their HIV treatment.