Real-World Efficacy of Midostaurin in Aggressive Systemic Mastocytosis
| Autor: | Oliwia Bachanek-Mitura, Witold Krupski, Tomasz Gromek, Aneta Szudy-Szczyrek, Justyna Szumiło, Michał Szczyrek, Grzegorz Helbig, Karolina Chromik, Marek Hus, Zuzanna Kanduła, Andrzej Mital |
|---|---|
| Jazyk: | angličtina |
| Rok vydání: | 2021 |
| Předmět: |
medicine.medical_specialty
Bone marrow infiltration tryptase lcsh:Medicine Tryptase mast cells Disease KIT D816V mutation Article 03 medical and health sciences chemistry.chemical_compound 0302 clinical medicine Internal medicine Medicine Secondary Acute Myeloid Leukemia Midostaurin Systemic mastocytosis biology business.industry lcsh:R aggressive systemic mastocytosis midostaurin General Medicine medicine.disease Transplantation chemistry 030220 oncology & carcinogenesis biology.protein Stem cell business 030215 immunology |
| Zdroj: | Journal of Clinical Medicine Journal of Clinical Medicine, Vol 10, Iss 1109, p 1109 (2021) Journal of Clinical Medicine; Volume 10; Issue 5; Pages: 1109 |
| ISSN: | 2077-0383 |
| Popis: | In April 2017 midostaurin was approved by the US Food and Drug Administration for the treatment of patients with aggressive systemic mastocytosis (ASM). So far, very limited real world data on its efficacy is available. Thirteen patients aged from 48 to 79 years, who received midostaurin in the early access program, were included in the study. Midostaurin was used both in first (n = 5) and subsequent lines of treatment (n = 8). The median duration of exposure was 9 months. Most patients (77%, n = 10) had a clinical improvement already as soon as the second month of therapy. Objective response was noted in 4 (50%) of eight evaluated patients. Among responders, we observed a decrease in serum tryptase level (median 74.14%) and bone marrow infiltration by mast cells (median 50%) in the sixth month of treatment. In one case, in the 10th month of treatment, allogenic stem cell transplantation was performed, achieving complete remission. Five patients died, three due to progression of disease, one in the course of secondary acute myeloid leukemia and one due to reasons not related to mastocytosis. Treatment is ongoing in seven patients. We found that midostaurin therapy is beneficial to patients with ASM. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|