Advanced
Systemic
Mastocytosis

Patients with Advanced SM experience organ damage which can lead to shortened overall survival1–3

Advanced SM is a rare clonal mast cell neoplasm4

Advanced SM has an estimated prevalence of ~1 in 100,000 and it commonly affects adults5

Drawing of a fictional patient’s medical file in a folder

Mast cell infiltration in Advanced SM may lead to debilitating symptoms and progressive organ dysfunction that significantly impacts daily life1,4

Patients with Advanced SM can suffer from severe and often life-limiting organ damage, including ascites, osteolytic bone lesions, liver dysfunction, weight loss, cytopenia, and hypersplenism1,6,7

Historical median OS for patients with Advanced SM2*

ASM (n=41)
41 months
41 months
SM-AHN (n=138)
24 months
24 months
MCL (n=4)
< 6 months
< 6 months

Figure adapted from Lim et al. 2009
*OS was examined in a retrospective study of 342 consecutive adult patients with SM at the Mayo Clinic between 1976 and 2007.2

Mechanism of Disease

Picture of KIT D816V mutation positive mast cell in bone marrow, with a close-up illustration of KIT D816V

Advanced SM is driven by KIT D816V mutation in ~95% of cases; however, until recently, there were no treatment options that selectively targeted this underlying mutation4–8

The KIT mutation constitutively activates downstream pathways regulating cellular functions including proliferation and survival of abnormal mast cells9,10

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Click here to learn more about AYVAKYT

About AYVAKYT

This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions. See section 4.8 of the product SmPC for how to report adverse reactions.1

References

  1. Gulen T, et al. J Intern Med. 2016;279(3):211–228.
  2. Lim KH, et al. Blood. 2009;113(23):5727–5736.
  3. Sperr WR, et al. Lancet Haematol. 2019;6(12):e638–e649.
  4. Mesa RA, et al. Cancer. 2022;128(20):3700–3708.
  5. Ungerstedt J, et al. Cancers (Basel). 2022;14(16):3942.
  6. Valent P, et al. Hemasphere. 2021;5(11):e646.
  7. Heiblig M, et al. Am J Hematol. 2024;99(11):2127–2139.

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