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Indolent ISMAdvanced SMPDGFRA GIST
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Indolent ISMAdvanced SMPDGFRA GIST

BURDEN OF ISM

Patient portrayal

ISM symptoms can create unpredictability and impact patients’ everyday lives1,2

Polypharmacy

Increased
polypharmacy2

Family and caregiver burden

Considerable family
and caregiver burden3

Reduced hours at work

Multiple HCP
visits per year2

In a Blueprint Medicines–sponsored survey (n=32)2*

72%

reported avoiding leaving home

56%

reported reducing working hours

28%

reported going on medical disability

ISM may lead to serious health consequences4-6

Symptoms often worsen over time4:

55% of patients with ISM have a higher frequency of symptoms
and
47.5% have greater severity of symptoms since receiving their diagnosis (N=40)†

Bone density concerns5:

Osteoporosis was observed in 66.9% of patients with ISM vs 34.3% of a similar patient
population without ISM‡

Risk of anaphylaxis6:

Nearly half of patients with ISM experience anaphylaxis§

In a Blueprint Medicines-sponsored survey of patients with ISM (n=37), 86% reported moderate to severe symptom burden despite taking a median of 3 over-the-counter medications and 2 prescription medications for their ISM symptoms.2*

*In the Blueprint Medicines–sponsored TouchStone SM Patient Survey, US adults with a self-reported SM diagnosis (N=56) completed an online survey of 100 items. An analysis was conducted in patients with ISM (n=37), including 32 patients with moderate to severe ISM (defined as an ISM-SAF TSS ≥28) and results were analyzed using descriptive statistics. These analyses were made from the TouchStone SM Patient Survey but have not been published.2

†Survey data were collected from 40 adults with ISM meeting the WHO diagnostic criteria, including the validated ISM-SAF and the 12-item Short-Form Health Survey. ISM burden was analyzed by comparing moderate to severe TSS scores with mild TSS scores using Kruskal–Wallis and Fisher’s exact tests.4

‡In a retrospective study of Mayo Clinic data (full system, 2005-2023), 203 patients with ISM were identified and matched 1:10 with 2030 patients without ISM on demographic and clinical characteristics (age, sex, race, BMI, smoking status, Quan-Charlson Comorbidity Index [CCI] score, and year of index [defined as first ISM diagnosis for ISM patients]), generating comparable cohorts with ISM diagnosis being the differentiating factor between groups.5

§As described by an expert-panel review of adult-onset mastocytosis (predominantly indolent population).6

BMI=body mass index; HCP=healthcare provider; ISM=indolent systemic mastocytosis; ISM-SAF=Indolent Systemic Mastocytosis-Symptom Assessment Form; SM=systemic mastocytosis; TSS=total symptom score; WHO=World Health Organization.

Assess disease control in ISM with the Mastocytosis Control Test (MCT)

The MCT is a validated, 5-question patient-reported outcomes tool designed to assess disease control and quality-of-life impact in adults with non-advanced mastocytosis, including ISM.7 It helps distinguish patients with poorly controlled disease from those with well-controlled disease. The MCT comes with a 1-page instruction sheet for healthcare providers to help interpret results.*

Mastocytosis Control Test (MCT)

Download the MCT

MCT Instructions and Scoring

Download the InstructionsDownload Link

*The MCT was developed at Charité by a team led by PD Dr. Frank Siebenhaar, PD Dr. Karsten Weller and Prof. Dr. Marcus Maurer with Dr. Cem Akin as a co-author. Blueprint Medicines (BPM) has secured a commercial license for distribution of this test to US healthcare providers only. If you have any questions on how this test was developed, please refer to the publication and/or BPM Medical Information at medinfo@blueprintmedicines.com or 1-888-BLU-PRNT (1-888-258-7768).7



Explore how AYVAKIT works

MOA
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References: 1. Pardanani A. Am J Hematol. 2023;98(7):1097-1116. 2. Data on file. Blueprint Medicines Corporation, Cambridge, MA. 2023. 3. Jennings SV et al. Immunol Allergy Clin North Am. 2018;38(3):505-525. 4. Zeiger RS et al. J Allergy Clin Immunol Pract. Published online October 28, 2024. doi:10.1016/j.jaip.2024.10.021 5. Data on file. Blueprint Medicines Corporation, Cambridge, MA. 2024. 6. Hartmann K et al. J Allergy Clin Immunol. 2016;137(1):35-45. 7. Siebenhaar F et al. J Allergy Clin Immunol Pract. 2025;13(3):647–657.

Expand +
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. If contraception requires estrogen, limit ethinyl estradiol to ≤20 mcg unless a higher dose is necessary.

To report suspected adverse reactions, contact Blueprint Medicines Corporation at 1-888-258-7768 or the FDA at 1-800-FDA-1088 or visit 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; November 2024.
  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.