# Between research and introduction to clinical routine—Experience with niraparib from the compassionate use program in Germany (NOGGO Register Analysis)

## TL;DR

This study evaluates the real-world use of niraparib in Germany for ovarian cancer, showing it is generally safe and effective despite some side effects.

## Contribution

The study provides real-world evidence of niraparib's safety and tolerability in compassionate use settings, complementing clinical trial data.

## Key findings

- Thrombocytopenia, fatigue, and nausea were the most common adverse events in patients receiving niraparib.
- Approximately half of the patients required dose adjustments due to side effects.
- Niraparib demonstrated favorable safety and tolerability in real-world clinical practice for recurrent ovarian cancer.

## Abstract

Ovarian cancer (OC) is frequently diagnosed at a late, advanced stage, resulting in poor survival outcomes. PARP inhibitors like niraparib have shown significant efficacy in high-grade OC, particularly in tumors with homologous recombination deficiency, including BRCA mutations. This study aimed to evaluate dose modifications, safety, tolerability, and the impact on quality of life associated with niraparib in real-world clinical practice.

This non-interventional, register-based study included patients with platinum-sensitive recurrent OC who received niraparib as part of the compassionate-use program (CUP) in Germany. Clinical baseline characteristics, treatment data, adverse events (AEs), and quality-of-life measures were collected both prospectively and retrospectively across 14 centers. Data analysis was performed using descriptive statistical methods.

Overall, 68 female patients were enrolled in the CUP register. Most patients had good performance status, with no significant comorbidities or concomitant medications. The most frequently reported AEs associated with niraparib were thrombocytopenia, fatigue, and nausea. Approximately half of patients required dose adjustments. AEs were less common in patients with normal physical examination findings, better ECOG performance status, and absence of comorbidities. Prior use of PARP inhibitors or previous treatment-related side effects increased the likelihood of AEs during niraparib therapy. The median treatment duration was 182 days, with disease progression being the most common reason for discontinuation.

Niraparib treatment within the German CUP demonstrated favorable safety and tolerability profiles, supporting its effectiveness in a real-world setting for patients with recurrent OC. These findings are consistent with results from clinical trials, further reinforcing the role of niraparib in this patient population.

The online version contains supplementary material available at 10.1007/s00404-025-08295-x.