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Oral Cyclophosphamide for Patients With Metastatic Castration-Resistant Prostate Cancer in a Scenario of Limited Health Care Resources

JCO Glob Oncol. 2025 Jan;11:e2400464. doi: 10.1200/GO-24-00464. Epub 2025 Jan 30.

ABSTRACT

PURPOSE: Prior noncontemporary studies showed that oral cyclophosphamide is an active treatment of metastatic castration-resistant prostate cancer (mCRPC). However, cyclophosphamide is currently underutilized in routine clinical practice given the lack of survival benefit and the emergence of more effective treatments.

METHODS: We retrospectively reviewed our institutional database to identify patients with mCRPC treated with cyclophosphamide. Prostate-specific antigen decrease ≥50% from baseline (PSA50) response was determined as the proportion of patients achieving a prostate-specific antigen (PSA) decline ≥50% from baseline. Radiographic responses and progression were evaluated by Prostate Cancer Working Group 3. Survival estimates used the Kaplan-Meier method, and correlations were made with Chi-square test for categorical variables.

RESULTS: From January 2011 to January 2023, 341 patients with mCRPC received oral cyclophosphamide at a tertiary cancer center in São Paulo, Brazil. The most common regimen (95%) was 100 mg once daily 21 days on, 7 days off. At prostate cancer diagnosis, the median age was 64.4 years (IQR, 59.4-70.8), 61.9% had metastatic de novo disease, and 55.5% had Gleason ≥8. The median number of previous treatment lines was three (IQR, 2-4). Any PSA decline was observed in 33.4%, and 13.2% had a PSA50 response. Median response duration was 2.1 months (IQR, 1.4-3.8). Ten patients (3%) were treated for ≥1 year. PSA50 response was associated with no prior docetaxel use and Eastern Cooperative Oncology Group performance status 0 or 1.

CONCLUSION: Oral cyclophosphamide is a feasible treatment option for patients with mCRPC, particularly in a scenario of limited health care resources.

PMID:39883895 | DOI:10.1200/GO-24-00464

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