TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS WITH MIDOSTAURIN: PRACTICAL GUIDANCE FOR OPTIMAL THERAPY AND MANAGEMENT Advanced systemic mastocytosis and midostaurin
Main Article Content
Keywords
systemic mastocytosis, midostaurin, guidance, management
Abstract
Systemic mastocytosis (SM) is a rare disease with a range of clinical presentations, and the vast majority of patients have a KIT D816V mutation that results in gain of function. The multikinase/KIT inhibitor midostaurin inhibits the D816V mutant, and has a well-established role in treatment of advanced SM. Even if considered the standard of therapy, some open questions remain on how to optimize the management of midostaurin in daily practice. The current review presents the opinions of a group of experts who met to discuss routine practice with the use of midostaurin in patients with advanced SM. The efficacy and safety of midostaurin in Phase 2 trials are overviewed, followed by practical guidance for optimal management of therapy and adverse events during therapy with midostaurin. Specific guidance is given for initiating therapy and evaluating response with midostaurin in terms of general assessment and laboratory, instrumental, pathological, and molecular exams. Special consideration is given to dose interruption, reduction, and discontinuation of therapy as well as adverse event management and supportive therapy. Patients should be informed about possible side effects and receive not only practical advice to avoid or limit them, but also antiemetic prophylaxis so that therapy with midostaurin can continue as long as clinical benefit is observed or until unacceptable toxicity occurs. Lastly, considerations on the use of midostaurin during the ongoing Covid-19 pandemic are made. The overall scope is to provide guidance that can be useful in daily practice for clinicians using midostaurin to treat patients with advanced SM.
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References
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2. Horny HP, Sotlar K and Valent P. Mastocytosis: state of the art. Pathobiology. 2007;74:121-132. https://doi.org/10.1159/000101711
3. Valent P, Akin C and Metcalfe DD. Mastocytosis: 2016 updated WHO classification and novel emerging treatment concepts. Blood. 2017;129:1420-1427. https://doi.org/10.1182/blood-2016-09-731893
4. Swerdlow SH. WHO classification of tumours of haematopoietic and lymphoid tissues 4th ed (revised). IARC Press; Lyon, France. 2017.
5. Pardanani A. Systemic mastocytosis in adults: 2021 Update on diagnosis, risk stratification and management. Am J Hematol. 2021;96:508-525.
https://doi.org/10.1002/ajh.26118
6. Martelli M, Monaldi C, De Santis S, Bruno S, Mancini M, Cavo M and Soverini S. Recent Advances in the Molecular Biology of Systemic Mastocytosis: Implications for Diagnosis, Prognosis, and Therapy. Int J Mol Sci. 2020;21:3987. https://doi.org/10.3390/ijms21113987
7. Schwaab J, Cabral do OHN, Naumann N, Jawhar M, Weiss C, Metzgeroth G, Schmid A, Lübke J, Reiter L, Fabarius A, Cross NCP, Sotlar K, Valent P, Kluin-Nelemans HC, Hofmann WK, Horny HP, Panse J and Reiter A. Importance of Adequate Diagnostic Workup for Correct Diagnosis of Advanced Systemic Mastocytosis. J Allergy Clin Immunol Pract. 2020;8:3121-3127. https://doi.org/10.1016/j.jaip.2020.05.005
8. Nagata H, Worobec AS, Oh CK, Chowdhury BA, Tannenbaum S, Suzuki Y and Metcalfe DD. Identification of a point mutation in the catalytic domain of the protooncogene c-kit in peripheral blood mononuclear cells of patients who have mastocytosis with an associated hematologic disorder. Proc Natl Acad Sci U S A. 1995;92:10560-10564. https://doi.org/10.1073/pnas.92.23.10560
9. Jawhar M, Schwaab J, Schnittger S, Meggendorfer M, Pfirrmann M, Sotlar K, Horny HP, Metzgeroth G, Kluger S, Naumann N, Haferlach C, Haferlach T, Valent P, Hofmann WK, Fabarius A, Cross NCP, Reiter A. Additional mutations in SRSF2, ASXL1 and/or RUNX1 identify a high-risk group of patients with KIT D816V(+) advanced systemic mastocytosis. Leukemia. 2016;30:136-143. https://doi.org/10.1038/leu.2015.284
10. Jawhar M, Schwaab J, Hausmann D, Clemens J, Naumann N, Henzler T, Horny HP, Sotlar K, Schoenberg SO, Cross NCP, Fabarius A, Hofmann WK, Valent P, Metzgeroth G and Reiter A. Splenomegaly, elevated alkaline phosphatase and mutations in the SRSF2/ASXL1/RUNX1 gene panel are strong adverse prognostic markers in patients with systemic mastocytosis. Leukemia 2016;30:2342-2350. https://doi.org/10.1038/leu.2016.190
11. Gilreath JA, Tchertanov L and Deininger MW. Novel approaches to treating advanced systemic mastocytosis. Clin Pharmacol. 2019;11:77-92. https://doi.org/10.2147/CPAA.S206615
12. Reiter A, George TI and Gotlib J. New developments in diagnosis, prognostication, and treatment of advanced systemic mastocytosis. Blood. 2020;135:1365-1376. https://doi.org/10.1182/blood.2019000932
13. Alvarez-Twose I, Matito A, Morgado JM, Sanchez-Munoz L, Jara-Acevedo M, Garcia-Montero A, Mayado A, Caldas C, Teodósio C, Muñoz-González JI, Mollejo M, Escribano L and Orfao A. Imatinib in systemic mastocytosis: a phase IV clinical trial in patients lacking exon 17 KIT mutations and review of the literature. Oncotarget. 2017;8:68950-68963. https://doi.org/10.18632/oncotarget.10711
14. Valent P, Cerny-Reiterer S, Hoermann G, Sperr WR, Mullauer L, Mannhalter C and Pehamberger H. Long-lasting complete response to imatinib in a patient with systemic mastocytosis exhibiting wild type KIT. Am J Blood Res. 2014;4:93-100.
15. Mannelli F. Catching the clinical and biological diversity for an appropriate therapeutic approach in systemic mastocytosis. Ann Hematol. 2021;100:337-344. https://doi.org/10.1007/s00277-020-04323-9
16. Piris-Villaespesa M and Alvarez-Twose I. Systemic Mastocytosis: Following the Tyrosine Kinase Inhibition Roadmap. Front Pharmacol. 2020;11:443. https://doi.org/10.3389/fphar.2020.00443
17. European Medicines Agency. Committee for Medicinal Products for Human Use (CHMP). Summary of opinion (post authorisation) Ayvakyt. https://www.ema.europa.eu/en/documents/smop/chmp-post-authorisation-summary-positive-opinion-ayvakyt-x-04-g_en.pdf (Accessed May 01, 2022).
18. Gotlib J, Gerds AT, Bose P, Castells MC, Deininger MW, Gojo I, Gundabolu K, Hobbs G, Jamieson C, McMahon B, Mohan SR, Oehler V, Oh S, Padron E, Pancari P, Papadantonakis N, Pardanani A, Podoltsev V, Rampal R, Ranheim E, Rein L, Snyder DS, Stein BL, Talpaz M, Thota S, Wadleigh M, Walsh K, Bergman MA and Sundar H. Systemic Mastocytosis, Version 2.2019, NCCN Clinical Practice Guidelines in Oncology. J Natl Compr Canc Netw. 2018;16:1500-1537. https://doi.org/10.6004/jnccn.2018.0088
19. Gotlib J, Kluin-Nelemans HC, George TI, Akin C, Sotlar K, Hermine O, Awan FT, Hexner E, Mauro MJ, Sternberg DW, Villeneuve M, Huntsman Labed A, Stanek EJ, Hartmann K, Horny HP, Valent P and Reiter A. Efficacy and Safety of Midostaurin in Advanced Systemic Mastocytosis. N Engl J Med. 2016;374:2530-2541. https://www.nejm.org/doi/10.1056/NEJMoa1513098
20. Gotlib J, Pardanani A, Akin C, Reiter A, George T, Hermine O, Kluin-Nelemans H, Hartmann K, Sperr WR, Brockow K, Schwartz LB, Orfao A, DeAngelo DJ, Arock M, Sotlar K, Horny HP, Metcalfe DD, Escribano L, Verstovsek S, Tefferi A and Valent P. International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) & European Competence Network on Mastocytosis (ECNM) consensus response criteria in advanced systemic mastocytosis. Blood. 2013;121:2393-2401. https://doi.org/10.1182/blood-2012-09-458521
21. DeAngelo DJ, George TI, Linder A, Langford C, Perkins C, Ma J, Westervelt P, Merker JD, Berube C, Coutre S, Liedtke M, Medeiros B, Sternberg D, Dutreix C, Ruffie PA, Corless C, Graubert TJ and Gotlib J. Efficacy and safety of midostaurin in patients with advanced systemic mastocytosis: 10-year median follow-up of a phase II trial. Leukemia. 2018;32:470-478. https://doi.org/10.1038/leu.2017.234
22. Midostaurin, Summary of Product Characteristics.
23. Gotlib J, Kluin-Nelemans HC, Akin C, Hartmann K, Valent P and Reiter A. Practical management of adverse events in patients with advanced systemic mastocytosis receiving midostaurin. Expert Opin Biol Ther. 2021:1-12. https://doi.org/10.1080/14712598.2021.1837109
24. Jawhar M, Schwaab J, Horny HP, Sotlar K, Naumann N, Fabarius A, Valent P, Cross NCP, Hofmann WK, Metzgeroth G and Reiter A. Impact of centralized evaluation of bone marrow histology in systemic mastocytosis. Eur J Clin Invest. 2016;46:392-397. https://doi.org/10.1111/eci.12607
25. Greiner G, Gurbisz M, Ratzinger F, Witzeneder N, Simonitsch-Klupp I, Mitterbauer-Hohendanner G, Mayerhofer M, Müllauer L, Sperr WR, Valent P and Hoermann G. Digital PCR: A Sensitive and Precise Method for KIT D816V Quantification in Mastocytosis. Clin Chem. 2018;64:547-555. https://doi.org/10.1373/clinchem.2017.277897
26. Arock M, Sotlar K, Akin C, Broesby-Olsen S, Hoermann G, Escribano L, Kristensen TK, Kluin-Nelemans HC, Hermine O, Dubreuil P, Sperr WR, Hartmann K, Gotlib J, Cross NCP, Haferlach T, Garcia-Montero A, Orfao A, Schwaab J, Triggiani M, Horny HP, Metcalfe DD, Reiter A and Valent P . KIT mutation analysis in mast cell neoplasms: recommendations of the European Competence Network on Mastocytosis. Leukemia. 2015;29:1223-1232. https://doi.org/10.1038/leu.2015.24
27. Criscuolo M, Fianchi L, Maraglino AME and Pagano L. Mastocytosis: One Word for Different Diseases. Oncol Ther. 2018;6:129-140. https://doi.org/10.1007/s40487-018-0086-2
28. Valent P, Akin C, Escribano L, Fodinger M, Hartmann K, Brockow K, Castells M, Sperr WR, Kluin-Nelemans HC, Hamdy NAT, Lortholary O, Robyn J, van Doormaal J, Sotlar K, Hauswirth AW, Arock M, Hermine O, Hellmann A, Triggiani M, Niedoszytko M, Schwartz LB, Orfao A, Horny HP and Metcalfe DD. Standards and standardization in mastocytosis: consensus statements on diagnostics, treatment recommendations and response criteria. Eur J Clin Invest. 2007;37:435-453.
https://doi.org/10.1111/j.1365-2362.2007.01807.x
29. Valent P, Akin C, Sperr WR, Horny HP, Arock M, Lechner K, Bennett JM and Metcalfe DD. Diagnosis and treatment of systemic mastocytosis: state of the art. Br J Haematol. 2003;122:695-717.
https://doi.org/10.1046/j.1365-2141.2003.04575.x
30. Gotlib J, Reiter A. ECNM 2020 Annual Meeting, Vienna, Austria.
31. Shomali W and Gotlib J. Response Criteria in Advanced Systemic Mastocytosis: Evolution in the Era of KIT Inhibitors. Int J Mol Sci. 2021;22:2983. https://doi.org/10.3390/ijms22062983
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Oct 29, 2022
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Papayannidis, C. (2022) “TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS WITH MIDOSTAURIN: PRACTICAL GUIDANCE FOR OPTIMAL THERAPY AND MANAGEMENT: Advanced systemic mastocytosis and midostaurin”, Mediterranean Journal of Hematology and Infectious Diseases, 14(1), p. e2022073. doi: 10.4084/MJHID.2022.073.
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