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Indolent ISMAdvanced SMPDGFRA GIST
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Indolent ISMAdvanced SMPDGFRA GIST

PATIENT PROFILES

Patient portrayal

Patient portrayals for indolent systemic mastocytosis (ISM)

The following patient portrayals are fictional examples of adult patients living with ISM and being treated with AYVAKIT that were developed from a review of the published literature. This information does not represent medical advice and individual results may vary. Healthcare providers should make all treatment decisions based on diagnostic criteria, clinical practice guidelines, prescribing information, individual patient circumstances, and their independent clinical judgment.

Review of this material does not substitute for a review of the Prescribing Information for AYVAKIT.

Jeff, a male indolent systemic mastocytosis patient portrayal profileJeff
Sondra, a female indolent systemic mastocytosis patient portrayal profileSondra

Scroll down to meet Jeff, a working father frustrated by a multiyear journey to ISM diagnosis, and Sondra, a mother of 3 living with ISM who is embarrassed by her ongoing symptoms.4-6,8,13

Hypothetical patients. Individual results may vary.

*Quotes have been fictionalized to align with hypothetical patient profiles.

Jeff

Sondra

Jeff, a 45-year-old male indolent systemic mastocytosis patient portrayal profile woodworking

Patient portrayal

Meet Jeff

CalendarAge: 451
CoupleMarried father of 2
Family statusForeman & softball coach
StethoscopeStruggle with ISM symptom burden leads to work and personal impacts5,6,13
ISM patient profile
Sondra, a female indolent systemic mastocytosis patient portrayal profile with her dog

Patient portrayal

Meet Sondra

CalendarAge: 471
CoupleMarried mother of 3
Family statusStay-at-home mom
StethoscopeStruggling to manage disease and symptoms4,13
ISM patient profile

*Quotes have been fictionalized to align with hypothetical patient profiles.

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Patient portrayal

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INDICATION

AYVAKIT® (avapritinib) is indicated for the treatment of adult patients with indolent systemic mastocytosis (ISM).

Limitations of Use: AYVAKIT is not recommended for the treatment of patients with ISM with platelet counts of <50 x 109/L.

IMPORTANT SAFETY INFORMATION
INDICATION & IMPORTANT SAFETY INFORMATION

Cognitive Effects—Cognitive adverse reactions can occur in patients receiving AYVAKIT and occurred in 7.8% of patients with ISM who received AYVAKIT + best supportive care (BSC) versus 7.0% of patients who received placebo + BSC; <1% were Grade 3. Depending on the severity, withhold AYVAKIT and then resume at the same dose, or permanently discontinue AYVAKIT.

Photosensitivity—AYVAKIT may cause photosensitivity reactions. In all patients treated with AYVAKIT in clinical trials (n=1049), photosensitivity reactions occurred in 2.5% of patients. Advise patients to limit direct ultraviolet exposure during treatment with AYVAKIT and for one week after discontinuation of treatment.

Embryo-Fetal Toxicity—AYVAKIT can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females and males of reproductive potential to use an effective contraception during treatment with AYVAKIT and for 6 weeks after the final dose. Advise women not to breastfeed during treatment with AYVAKIT and for 2 weeks following the final dose.

Adverse Reactions—The most common adverse reactions (≥10%) in patients with ISM were eye edema, dizziness, peripheral edema, and flushing.

Drug Interactions—Avoid coadministration of AYVAKIT with strong or moderate CYP3A inhibitors or inducers.

To report suspected adverse reactions, contact Blueprint Medicines Corporation at 1-888-258-7768 or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please click here to see the full Prescribing Information for AYVAKIT.

References:

  1. AYVAKIT [prescribing information]. Cambridge, MA: Blueprint Medicines Corporation; May 2023.
  2. Kristensen T et al. Am J Hematol. 2014;89(5):493-498.
  3. Garcia-Montero AC et al. Blood. 2006;108(7):2366-2372.
  4. Ungerstedt J et al. Cancers. 2022;14(16):3942.
  5. Pardanani A. Am J Hematol. 2023;98(7):1097-1116.
  6. Data on file. Blueprint Medicines Corporation, Cambridge, MA. 2023.
  7. Gülen T et al. J Intern Med. 2016;279(3):211-228.
  8. Theoharides TC et al. N Engl J Med. 2015;373(2):163-172.
  9. Gotlib J et al. NEJM Evidence. 2023;2(6). Published online May 23, 2023. doi:10.1056/EVIDoa2200339
  10. Gilreath JA et al. Clin Pharmacol. 2019;11:77-92.
  11. Evans EK et al. Sci Transl Med. 2017;9(414):eaao1690.
  12. Padilla B et al. Orphanet J Rare Dis. 2021;16(1):434.
  13. van Anrooij B et al. Allergy. 2016;71(11):1585-1593.
  14. WHO Classification of Tumours Editorial Board. Haematolymphoid tumours [Internet]. Lyon (France): International Agency for Research on Cancer; 2024 [cited April 24, 2024]. (WHO Classification of Tumours Series, 5th ed.; vol. 11). Available from: https://tumourclassification.iarc.who.int/chapters/63
  15. Dranitsaris G et al. J Oncol Pharm Pract. Published online December 27, 2023. doi:10.1177/10781552231221149
  16. Siebenhaar F et al. Immunol Allergy Clin North Am. 2014;34(2):433-447.
  17. Jennings SV et al. Immunol Allergy Clin North Am. 2018;38(3):505-525.
  18. Akin C, ed. Mastocytosis: A Comprehensive Guide. Springer; 2020.