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Pharmacist Focused Strategies for Systemic Mastocytosis

CE

Systemic Mastocytosis: Pharmacist-Focused Strategies to Improve Outcomes

Pharmacists: 1.00 contact hour (0.1 CEUs)

Released: May 22, 2025

Expiration: May 21, 2026

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References

  1. Akin C, Siebenhaar F, Wechsler JB, et al. Detecting changes in mast cell numbers versus activation in human disease: a roadblock for current biomarkers? J Allergy Clin Immunol Pract. 2024;12:1727-1737.
  2. Keow J, Chin-Yee B, Hsia CC, et al. Urticaria pigmentosa and systemic mastocytosis. Clin Case Rep. 2023;11:e8302.
  3. Ustun C, Keklik Karadag F, Linden MA, et al. Systemic mastocytosis: current status and challenges in 2024. Blood Adv. 2025;9:2048-2062.
  4. Gotlib J, Gerds AT, Abdelmessieh P, et al. NCCN Guidelines® Insights: systemic mastocytosis, Version 3.2024. J Natl Compr Canc Netw. 2024;22:e240030.
  5. Brockow K. Epidemiology, prognosis, and risk factors in mastocytosis. Immunol Allergy Clin North Am. 2014;34:283-295.
  6. Jennings S, Russell N, Jennings B, et al. The Mastocytosis Society survey on mast cell disorders: patient experiences and perceptions. J Allergy Clin Immunol Pract. 2014;2:70-76.
  7. Jennings SV, Slee VM, Zack RM, et al. Patient perceptions in mast cell disorders. Immunol Allergy Clin North Am. 2018;38:505-525.
  8. Rüfer A, Nilius H, Hermine O, et al. Prognostic impact of expression of CD2, CD25, and/or CD30 in/on mast cells in systemic mastocytosis: a registry study of the European Competence Network on Mastocytosis. Leukemia. 2025;39:675-683.
  9. Cilloni D, Maffeo B, Savi A, et al. Detection of KIT mutations in systemic mastocytosis: how, when, and why. Int J Mol Sci. 2024;25:10885.
  10. Hartmann K, Escribano L, Grattan C, et al. Cutaneous manifestations in patients with mastocytosis: consensus report of the European Competence Network on Mastocytosis; the American Academy of Allergy, Asthma & Immunology; and the European Academy of Allergology and Clinical Immunology. J Allergy Clin Immunol. 2016;137:35-45.
  11. Veitch S, Radia DH. Mastocytosis demystified. Hematology Am Soc Hematol Educ Program. 2023;2023:396-406.
  12. National Comprehensive Cancer Network. Clinical practice guidelines in oncology: systemic mastocytosis. v.1.2025 nccn.org. Accessed May 15, 2025.
  13. Valent P, Akin C, Hartmann K, et al. Updated diagnostic criteria and classification of mast cell disorders: a consensus proposal. Hemasphere. 2021;5:e646.
  14. Zuberbier T, Abdul Latiff AH, Abuzakouk M, et al. The international EAACI/GA²LEN/EuroGuiDerm/APAAACI guideline for the definition, classification, diagnosis, and management of urticaria. Allergy. 2022;77:734-766.
  15. Montelukast sodium [prescribing information]. Whitehouse Station, NJ: Merck; 2020.
  16. Avapritnib [prescribing information]. Cambridge, MA: Blueprint Medicines; 2024.
  17. Midostaurin [prescribing information]. East Hanover, NJ: Novartis; 2023.
  18. Imatinib mesylate [prescribing information]. East Hanover, NJ: Novartis; 2022.
  19. Tremblay D, Wagner NE, Mascarenhas J. Management of advanced systemic mastocytosis: clinical challenges. J Blood Med. 2024;15:421-433.
  20. Gotlib J, Castells M, Elberink HO, et al. Avapritinib versus placebo in indolent systemic mastocytosis. NEJM Evid. 2023;2:EVIDoa2200339.
  21. Castells M. Long-term quality of life and safety in patients with indolent systemic mastocytosis treated with avapritnib in the PIONEER Study. Presented at: AAAI/WAO Joint Congress; February 28 - March 3, 2025. Abstract 534.
  22. Reiter A, Gotlib J, Álvarez-Twose I, et al. Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis. Leukemia. 2022;36:2108-2120.
  23. Reiter A, Schwaab J, DeAngelo DJ, et al. Efficacy and safety of avapritinib in previously treated patients with advanced systemic mastocytosis. Blood Adv. 2022;6:5750-5762.
  24. Tashi T. Elenestinib, an investigational, next generation KIT D816V inhibitor, reduces mast cell burden, improves symptoms, and has a favorable safety profile in patients with indolent systemic mastocytosis: analysis of the HARBOR Trial. Presented at: 65th American Society of Hematology Meeting and Exposition; December 9-12, 2023. Abstract 76.
  25. Rein L, Modena B, Siebenhaar F, et al. Symptom-focused results from SUMMIT part 1: an ongoing, randomized, double-blind, placebo-controlled phase 2 clinical trial of bezuclastinib in adult patients with nonadvanced systemic mastocytosis. Presented at: European Hematology Association Conference 2024; June 13-16, 2024. Abstract P1055.
  26. DeAngelo, D. APEX Part 1: updated assessment of bezuclastinib (CGT9486), a selective KIT D816V tyrosine kinase inhibitor, in patients with advanced systemic mastocytosis (AdvSM). Presented at: European Hematology Association Conference 2024; June 13-16, 2024. Abstract 659.
  27. Australasian Society of Clinical Immunology and Allergy. Anaphylaxis checklist: pharmacists. allergy.org.au/hp/anaphylaxis/anaphylaxis-checklist-for-pharmacists. Accessed May 13, 2025.
  28. Gotlib J, Gerds AT, Bose P, et al. Systemic mastocytosis, Version 2.2019, NCCN clinical practice guidelines in oncology. J Natl Compr Canc Netw. 2018;16:1500-1537.
  29. Mesa RA, Sullivan EM, Dubinski D, et al. Patient-reported outcomes among patients with systemic mastocytosis in routine clinical practice: results of the TouchStone SM Patient Survey. Cancer. 2022;128:3691-3699.
  30. Sherman JJ, Davis L, Daniels K. Addressing the polypharmacy conundrum. US Pharm. 2017;42:HS-14-HS-20.
  31. Akin C, Gotlib J, Alvarez-Twose, I, et al. Reductions in polypharmacy for patients with indolent systemic mastocytosis on avapritinib. J Allergy Clin Immunol. 2024;153:AB225.
  32. Hamilton MJ, Greene LW, Madigan LM, et al. Case report: multidisciplinary management of a patient with indolent systemic mastocytosis and refractory symptoms. Front Allergy. 2024;5:1401187.

You are doing a medication reconciliation, and you identify that the patient is receiving treatment for osteoporosis, migraine, and frequent diarrhea.

Based on this information, which is the most appropriate statement regarding the need to evaluate the patient for SM?

Your patient has indolent SM with KIT D816V mutation and initially found relief for itching and diarrhea with antimediator therapies, but after 6 months the symptoms are worsening. 

Based on the PIONEER trial, which is the most accurate information you can give your patient on the duration of symptom benefits observed with the selective KIT inhibitor avapritinib? 

In educating patients with SM on epinephrine use, which of the following represents correct information that you could provide when discussing their anaphylaxis risk?

How often do you educate patients who have an epinephrine prescription on the need for a treatment plan that outlines when epinephrine should be used?