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Apatinib combined with temozolomide in diffuse midline glioma: a novel and effective therapy
Yu-An Li1, Chuan Zhao1, Jing-Jing Ge1
1Department of Neuro-Oncology, Sanbo Brain Hospital, Capital Medical University, Beijing, China.
Purpose:
Diffuse midline glioma (DMG), H3 K27M-mutant is a type of diffuse high-grade glioma that occurs in the brain midline carrying an extremely poor prognosis under the best efforts of surgery, radiation, and other therapies. For better therapy, we explored the efficacy and toxicity of a novel therapy that combines apatinib and temozolomide in DMG.
Methods:
A retrospective analysis of 32 patients with DMG who underwent apatinib plus temozolomide treatment was performed. Apatinib was given 500 mg in adults, 250 mg in pediatric patients once daily. Temozolomide was administered at 200 mg/m2/d according to the standard 5/28 days regimen. The main clinical data included basic information of patients, radiological and pathological characteristics of tumors, treatment, adverse reactions, prognosis.
Results:
The objective response rate was 24.1%, and the disease control rate was 79.3%. The median PFS of all patients was 5.8 months, and median OS was 10.3 months. A total of 236 cycles of treatment were available for safety assessment and the toxicity of the combination therapy was relatively well tolerated. The most common grade 3 toxicities were myelosuppression including leukopenia (5.08%), neutropenia (4.24%), lymphopenia (2.12%), thrombocytopenia (1.69%) and anemia (1.27%). Grade 4 toxicities included neutropenia (2.12%), thrombocytopenia (2.12%) and proteinuria (1.69%). All the adverse events were relieved after symptomatic treatment or dose reduction.
Conclusions:
Apatinib plus temozolomide could be an effective regimen with manageable toxicities and favorable efficacy and may outperform temozolomide monotherapy, particularly in newly diagnosed adults with tumors located outside the pons. The novel therapy deserves further investigation in adult DMG patients.
Insights
This study shows that combining apatinib and temozolomide is an effective treatment for diffuse midline glioma (DMG), offering better outcomes than temozolomide alone. The novel therapy demonstrated manageable side effects in patients with this aggressive brain tumor.
Area of Science:
- Neuro-oncology
- Clinical Pharmacology
- Cancer Therapeutics
Background:
- Diffuse midline glioma (DMG), H3 K27M-mutant, is an aggressive brain tumor with a poor prognosis.
- Current treatments including surgery, radiation, and chemotherapy offer limited efficacy for DMG.
Purpose of the Study:
- To evaluate the efficacy and toxicity of a combination therapy using apatinib and temozolomide in patients with DMG.
- To explore a novel therapeutic approach for improving outcomes in diffuse midline glioma.
Main Methods:
- Retrospective analysis of 32 patients with DMG treated with apatinib and temozolomide.
- Apatinib dosage: 500 mg (adults) or 250 mg (pediatric) daily; Temozolomide: 200 mg/m²/day (5/28 days regimen).
- Data collected on patient demographics, tumor characteristics, treatment response, adverse events, and survival.
Main Results:
- Objective response rate (ORR) was 24.1%; disease control rate (DCR) was 79.3%.
- Median progression-free survival (PFS) was 5.8 months; median overall survival (OS) was 10.3 months.
- Combination therapy was well-tolerated; common toxicities included myelosuppression (leukopenia, neutropenia, lymphopenia, thrombocytopenia, anemia) and proteinuria, manageable with dose adjustments.
Conclusions:
- Apatinib plus temozolomide demonstrates favorable efficacy and manageable toxicity in DMG patients.
- This combination may offer superior outcomes compared to temozolomide monotherapy, especially in newly diagnosed adult patients with non-pontine tumors.
- Further investigation of this novel therapy in adult DMG is warranted.
