Real-World Efficacy of Midostaurin in Aggressive Systemic Mastocytosis
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 earl...
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MDPI AG
2021-03-01
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| Series: | Journal of Clinical Medicine |
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| Online Access: | https://www.mdpi.com/2077-0383/10/5/1109 |
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| author | Aneta Szudy-Szczyrek Oliwia Bachanek-Mitura Tomasz Gromek Karolina Chromik Andrzej Mital Michał Szczyrek Witold Krupski Justyna Szumiło Zuzanna Kanduła Grzegorz Helbig Marek Hus |
| author_facet | Aneta Szudy-Szczyrek Oliwia Bachanek-Mitura Tomasz Gromek Karolina Chromik Andrzej Mital Michał Szczyrek Witold Krupski Justyna Szumiło Zuzanna Kanduła Grzegorz Helbig Marek Hus |
| author_sort | Aneta Szudy-Szczyrek |
| collection | DOAJ |
| description | 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 (<i>n</i> = 5) and subsequent lines of treatment (<i>n</i> = 8). The median duration of exposure was 9 months. Most patients (77%, <i>n</i> = 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. |
| first_indexed | 2024-03-09T05:08:05Z |
| format | Article |
| id | doaj.art-6ed0147c65ba447c9f7857c80a09b624 |
| institution | Directory Open Access Journal |
| issn | 2077-0383 |
| language | English |
| last_indexed | 2024-03-09T05:08:05Z |
| publishDate | 2021-03-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Journal of Clinical Medicine |
| spelling | doaj.art-6ed0147c65ba447c9f7857c80a09b6242023-12-03T12:53:05ZengMDPI AGJournal of Clinical Medicine2077-03832021-03-01105110910.3390/jcm10051109Real-World Efficacy of Midostaurin in Aggressive Systemic MastocytosisAneta Szudy-Szczyrek0Oliwia Bachanek-Mitura1Tomasz Gromek2Karolina Chromik3Andrzej Mital4Michał Szczyrek5Witold Krupski6Justyna Szumiło7Zuzanna Kanduła8Grzegorz Helbig9Marek Hus10Chair and Department of Haematooncology and Bone Marrow Transplantation, Medical University of Lublin Staszica Street 11, 20-081 Lublin, PolandChair and Department of Haematooncology and Bone Marrow Transplantation, Medical University of Lublin Staszica Street 11, 20-081 Lublin, PolandChair and Department of Haematooncology and Bone Marrow Transplantation, Medical University of Lublin Staszica Street 11, 20-081 Lublin, PolandDepartment of Hematology and Bone Marrow Transplantation, Medical University of Silesia in Katowice, 40-032 Katowice, PolandDepartment of Hematology and Transplantology, Medical University of Gdańsk, 80-211 Gdańsk, PolandChair and Department of Pneumonology, Oncology and Allergology, Medical University of Lublin, 20-090 Lublin, PolandII Department of Medical Radiology, Medical University of Lublin, 20-081 Lublin, PolandChair and Department of Clinical Pathomorphology, Medical University of Lublin, 20-090 Lublin, PolandDepartment of Hematology and Bone Marrow Transplantation, University of Medical Sciences in Poznan, 61-001 Poznań, PolandDepartment of Hematology and Bone Marrow Transplantation, Medical University of Silesia in Katowice, 40-032 Katowice, PolandChair and Department of Haematooncology and Bone Marrow Transplantation, Medical University of Lublin Staszica Street 11, 20-081 Lublin, PolandIn 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 (<i>n</i> = 5) and subsequent lines of treatment (<i>n</i> = 8). The median duration of exposure was 9 months. Most patients (77%, <i>n</i> = 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.https://www.mdpi.com/2077-0383/10/5/1109aggressive systemic mastocytosismidostaurinmast cellstryptase<i>KIT D816V</i> mutation |
| spellingShingle | Aneta Szudy-Szczyrek Oliwia Bachanek-Mitura Tomasz Gromek Karolina Chromik Andrzej Mital Michał Szczyrek Witold Krupski Justyna Szumiło Zuzanna Kanduła Grzegorz Helbig Marek Hus Real-World Efficacy of Midostaurin in Aggressive Systemic Mastocytosis Journal of Clinical Medicine aggressive systemic mastocytosis midostaurin mast cells tryptase <i>KIT D816V</i> mutation |
| title | Real-World Efficacy of Midostaurin in Aggressive Systemic Mastocytosis |
| title_full | Real-World Efficacy of Midostaurin in Aggressive Systemic Mastocytosis |
| title_fullStr | Real-World Efficacy of Midostaurin in Aggressive Systemic Mastocytosis |
| title_full_unstemmed | Real-World Efficacy of Midostaurin in Aggressive Systemic Mastocytosis |
| title_short | Real-World Efficacy of Midostaurin in Aggressive Systemic Mastocytosis |
| title_sort | real world efficacy of midostaurin in aggressive systemic mastocytosis |
| topic | aggressive systemic mastocytosis midostaurin mast cells tryptase <i>KIT D816V</i> mutation |
| url | https://www.mdpi.com/2077-0383/10/5/1109 |
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