Related Experiment Video
Updated: Feb 4, 2026

Human Liver Microphysiological System for Assessing Drug-Induced Liver Toxicity In Vitro
Published on: January 31, 2022
Niraparib - A promising drug with hematological toxicity
Naga Sk Patibandla1, Dulabh K Monga2
11 Department of Internal Medicine, Allegheny General Hospital, Allegheny Health Network, Pittsburgh, PA, USA.
Abstract:
Ovarian cancer is the second most common and the most lethal gynecological malignancy in the western world. Unfortunately, there are lack of methods for early screening and diagnosis of the disease. Because of this, most of the cases are diagnosed at an advanced stage and have poor prognosis. The standard treatment of ovarian cancer is maximal cytoreductive surgical debulking followed by platinum-based chemotherapy. There are new molecular agents available for maintenance therapy of ovarian cancer including anti-angiogenic therapies, poly adenosine diphosphate ribose polymerase inhibitors, inhibitors of growth factor signaling, or folate receptor inhibitors, as well as several immunotherapeutic approaches. Niraparib is a poly adenosine diphosphate ribose polymerase inhibitor that has shown to be clinically effective as maintenance therapy in patients with platinum sensitive, recurrent ovarian cancer. Studies have shown the median duration of progression-free survival was significantly longer among those receiving niraparib than among those receiving placebo, regardless of presence or absence of BRCA gene mutations or homologous recombination deficiency status. Studies have shown that treatment-emergent Grade 3 or Grade 4 hematological events were observed in patients receiving niraparib including thrombocytopenia (33.8%), anemia (25.3%) and neutropenia (19.6%). Most of the hematological laboratory abnormalities occurred within the first three treatment cycles. After dose adjustment, the incidence of hematological abnormalities was infrequent beyond cycle 3. We are reporting two cases of Grade III/IV neutropenia and thrombocytopenia in patients treated with niraparib in our institution. Unfortunately, one of the patients succumbed to septic shock secondary to right lower lobe pneumonia while severely neutropenic. The second patient's blood counts improved after discontinuing the medication and with supportive transfusions during the hospitalization.
Insights
Niraparib effectively extends progression-free survival in recurrent ovarian cancer. However, severe neutropenia and thrombocytopenia can occur, necessitating careful monitoring and dose adjustment.
Area of Science:
- Gynecologic Oncology
- Pharmacology
- Hematology
Background:
- Ovarian cancer is a leading cause of gynecologic cancer mortality due to late diagnosis.
- Standard treatment involves surgery and platinum-based chemotherapy.
- Maintenance therapies, including poly (ADP-ribose) polymerase (PARP) inhibitors, offer new treatment avenues.
Observation:
- Niraparib, a PARP inhibitor, demonstrates efficacy in maintaining progression-free survival for recurrent ovarian cancer patients.
- Clinical trials show improved progression-free survival with niraparib, irrespective of BRCA mutation or homologous recombination deficiency status.
- Significant Grade 3 or 4 hematological events, including thrombocytopenia, anemia, and neutropenia, are observed with niraparib treatment.
Findings:
- Hematological abnormalities commonly occur within the first three cycles of niraparib therapy.
- Dose adjustments can reduce the incidence of hematological events beyond the third cycle.
- This report details two cases of severe neutropenia and thrombocytopenia in patients receiving niraparib, with one fatality due to septic shock.
Implications:
- Niraparib is a valuable option for ovarian cancer maintenance therapy.
- Close hematological monitoring and prompt management of adverse events are crucial for patient safety.
- Further research may explore strategies to mitigate niraparib-induced hematotoxicity.
Related Concept Videos
Toxic Reactions: Overview
Toxicity falls into two primary categories: local and systemic.
Local toxicity appears at the exposure site, such as protein denaturation caused by caustic substances.
In contrast, systemic toxicity requires the toxic agent's absorption and distribution,...
Pharmacokinetics: Drug–Drug Interactions
Bioequivalence of Drugs: Drugs with Multiple Indications
FDA Approved Drugs: Changes to Approved Drugs
Factors Influencing Drug Absorption: Drug Dissolution
Factors Affecting Protein-Drug Binding: Drug-Related Factors
One crucial factor in drug-protein binding is the drug's lipophilicity or its affinity for fat. More lipophilic drugs tend to have higher binding extents. For example, highly lipophilic drugs like cloxacillin exhibit substantial protein binding, with as much as 95% of the drug binding to proteins. In...

