AYVAKIT® (avapritinib) resources for you
and your patients with Advanced SM
and your patients with Advanced SM
Dr Ruben Mesa from MD Anderson Cancer Center discusses the journey from suspecting Advanced SM to making a diagnosis.
Dr Ruben Mesa from MD Anderson Cancer Center reviews information about the efficacy and safety of AYVAKIT, as demonstrated in the PATHFINDER and EXPLORER clinical trials.
Dr James McCloskey from Hackensack Meridian Health reviews information about managing patients who have started treatment with AYVAKIT.
POPUP 1 : CONTENT HERE
POPUP 2 : CONTENT HERE
POPUP 3 : CONTENT HERE
Dr Ruben Mesa from MD Anderson Cancer Center discusses the journey from suspecting Advanced SM to making a diagnosis.
TranscriptVideo Transcript
Suspecting and Diagnosing Advanced Systemic Mastocytosis (SM)
Dr Mesa Voice-over:
Hello. I’m Dr Ruben Mesa, and in this video, we’re talking about suspecting and diagnosing Advanced Systemic Mastocytosis.
Advanced Systemic Mastocytosis (or Advanced SM) is a clonal mast cell neoplasm caused by the KIT D816V mutation in approximately 95% of cases.
People living with Advanced SM can experience significant symptom burden, including organ damage, and impact on their quality of life.
Patients with Advanced SM may also have shortened overall survival.
In fact, one study showed that the median overall survival was 3.5 years for patients with aggressive systemic mastocytosis (or ASM), 2 years for patients with systemic mastocytosis with an associated hematological neoplasm (or SM-AHN), and less than 6 months for patients with mast cell leukemia (or MCL).
So, the sooner Advanced SM can be diagnosed, the sooner we can determine if treatment may be appropriate for our patients.
The challenge to diagnosing Advanced SM comes from its symptoms. They can be heterogeneous or nonspecific, so one patient may end up presenting to many different healthcare specialists, which can prolong a diagnosis.
In fact, the median time from symptom onset to diagnosis for patients with Advanced SM is generally around 3 years.
Knowing the symptoms of Advanced SM may help shorten a patient’s time to diagnosis.
Common mast cell mediator symptoms of Advanced SM include maculopapular rash, gastrointestinal distress, and life-threatening anaphylaxis. Additionally, patients can experience organ damage and related symptoms, as shown here.
Accurately diagnosing Advanced SM is a multistep process.
The diagnostic workup for suspected Advanced SM includes a bone marrow biopsy with mast cell immunophenotyping, a serum tryptase test, and KIT D816V mutation testing.
Advanced SM may be overlooked or missed in some patients with suspected myeloid neoplasms.
Knowing this, an incidental KIT mutation finding should prompt a full diagnostic workup.
Generally, in patients where Advanced SM is suspected, it is recommended to perform high-sensitivity KIT D816V testing, which refers to assays with sensitivities of less than 1%. Next- generation sequencing panels can exhibit low sensitivity and fail to detect KIT D816V.
Looking at the diagnostic criteria for SM, the World Health Organization (or WHO) requires presence of 1 major criterion AND at least 1 minor criterion—OR, in the absence of 1 major criterion, presence of at least 3 minor criteria is sufficient to make a diagnosis.
Additionally, for an Advanced SM diagnosis, criteria for one of the three subtypes must be met.
I want to thank you for your time.
Please take a moment to familiarize yourselves with the Important Safety Information associated with AYVAKIT.
Important Safety Information Voice-over:
AYVAKIT® (avapritinib) is indicated for the treatment of adult patients with Advanced SM including patients with aggressive systemic mastocytosis, systemic mastocytosis with an associated hematological neoplasm, and mast cell leukemia.
Limitations of Use: AYVAKIT is not recommended for the treatment of patients with Advanced SM with platelet counts of less than 50,000 per microliter.
IMPORTANT SAFETY INFORMATION
There are no contraindications for AYVAKIT.
Serious intracranial hemorrhage (ICH) may occur with AYVAKIT treatment; fatal events occurred in less than 1% of patients. Overall, ICH (for example, subdural hematoma, ICH, and cerebral hemorrhage) occurred in 2.9% of 749 patients who received AYVAKIT. In Advanced SM patients who received AYVAKIT at 200 milligrams daily, ICH occurred in 2 of 75 patients (2.7%) who had platelet counts greater than or equal to 50,000 per microliter prior to initiation of therapy and in 3 of 80 patients (3.8%) regardless of platelet counts. Monitor patients closely for risk of ICH including those with thrombocytopenia, vascular aneurysm or a history of ICH or cerebrovascular accident within the prior year. Permanently discontinue AYVAKIT if ICH of any grade occurs. A platelet count must be performed prior to initiating therapy.
AYVAKIT is not recommended in Advanced SM patients with platelet counts less than 50,000 per microliter. Following treatment initiation, platelet counts must be performed every 2 weeks for the first 8 weeks. After 8 weeks of treatment, monitor platelet counts every 2 weeks or as clinically indicated based on platelet counts. Manage platelet counts of less than 50,000 per microliter by treatment interruption or dose reduction.
Cognitive adverse reactions can occur in patients receiving AYVAKIT. Cognitive adverse reactions occurred in 39% of 749 patients and in 28% of 148 Advanced SM patients (3% were greater than or equal to Grade 3). Memory impairment occurred in 16% of patients; all events were Grade 1 or 2. Cognitive disorder occurred in 10% of patients; less than 1% of these events were Grade 3. Confusional state occurred in 6% of patients; less than 1% of these events were Grade 3. Other events occurred in less than 2% of patients. Depending on the severity, withhold AYVAKIT and then resume at same dose or at a reduced dose upon improvement, or permanently discontinue.
AYVAKIT may cause photosensitivity reactions. In all 803 patients treated with AYVAKIT in clinical trials, 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.
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 method of contraception during treatment with AYVAKIT and for 6 weeks after the final dose of AYVAKIT. Advise women not to breastfeed during treatment with AYVAKIT and for 2 weeks after the final dose.
The most common adverse reactions (greater than or equal to 20%) were edema, diarrhea, nausea, and fatigue/asthenia.
Avoid coadministration of AYVAKIT with strong and moderate CYP3A inhibitors. If coadministration with a moderate CYP3A inhibitor cannot be avoided, reduce dose of AYVAKIT. Avoid coadministration of AYVAKIT with strong and moderate CYP3A 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 see the full Prescribing Information for AYVAKIT at AYVAKITHCP.com/advsm.
The downloadable materials below are here to provide additional support and information for you and your patients on AYVAKIT.
Dosing and Patient Management Guide for Advanced SM
Product Order Sheet for Advanced SM
Access and Reimbursement Guide
Product Brochure for Advanced SM
Identifying Advanced SM
Patient Brochure
for Advanced SM
These materials are intended for digital use only. If you decide to print them, please be sure to print and attach a copy of the full Prescribing Information as well.
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INDICATION
INDICATION & IMPORTANT SAFETY INFORMATION
AYVAKIT® (avapritinib) is indicated for the treatment of adult patients with Advanced SM (AdvSM) including patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL).
Limitations of Use: AYVAKIT is not recommended for the treatment of patients with AdvSM with platelet counts of <50 x 109/L.
IMPORTANT SAFETY INFORMATION
Intracranial Hemorrhage—Serious intracranial hemorrhage (ICH) may occur with AYVAKIT treatment; fatal events occurred in <1% of patients. Overall, ICH (eg, subdural hematoma, ICH, and cerebral hemorrhage) occurred in 2.9% of 749 patients who received AYVAKIT. In AdvSM patients who received AYVAKIT at 200 mg daily, ICH occurred in 2 of 75 patients (2.7%) who had platelet counts ≥50 x 109/L prior to initiation of therapy and in 3 of 80 patients (3.8%) regardless of platelet counts. Monitor patients closely for risk of ICH including those with thrombocytopenia, vascular aneurysm or a history of ICH or cerebrovascular accident within the prior year. Permanently discontinue AYVAKIT if ICH of any grade occurs. A platelet count must be performed prior to initiating therapy. AYVAKIT is not recommended in AdvSM patients with platelet counts <50 x 109/L. Following treatment initiation, platelet counts must be performed every 2 weeks for the first 8 weeks. After 8 weeks of treatment, monitor platelet counts every 2 weeks or as clinically indicated based on platelet counts. Manage platelet counts of <50 x 109/L by treatment interruption or dose reduction.
Cognitive Effects—Cognitive adverse reactions can occur in patients receiving AYVAKIT. Cognitive adverse reactions occurred in 39% of 749 patients and in 28% of 148 AdvSM patients (3% were Grade ≥3). Memory impairment occurred in 16% of patients; all events were Grade 1 or 2. Cognitive disorder occurred in 10% of patients; <1% of these events were Grade 3. Confusional state occurred in 6% of patients; <1% of these events were Grade 3. Other events occurred in <2% of patients. Depending on the severity, withhold AYVAKIT and then resume at same dose or at a reduced dose upon improvement, or permanently discontinue.
Photosensitivity—AYVAKIT may cause photosensitivity reactions. In all patients treated with AYVAKIT in clinical trials (n=803), 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 method of contraception during treatment with AYVAKIT and for 6 weeks after the final dose of AYVAKIT. Advise women not to breastfeed during treatment with AYVAKIT and for 2 weeks after the final dose.
Adverse Reactions—The most common adverse reactions (≥20%) were edema, diarrhea, nausea, and fatigue/asthenia.
Drug Interactions—Avoid coadministration of AYVAKIT with strong and moderate CYP3A inhibitors. If coadministration with a moderate CYP3A inhibitor cannot be avoided, reduce dose of AYVAKIT. Avoid coadministration of AYVAKIT with strong and moderate CYP3A 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.
INDICATION
AYVAKIT® (avapritinib) is indicated for the treatment of adult patients with Advanced SM (AdvSM) including patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL).
Limitations of Use: AYVAKIT is not recommended for the treatment of patients with AdvSM with platelet counts of <50 x 109/L.
References:
- AYVAKIT [prescribing information]. Cambridge, MA: Blueprint Medicines Corporation; March 2023.
- Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Systemic Mastocytosis V. 2.2022. © National Comprehensive Cancer Network, Inc. 2022. All rights reserved. Accessed February 15, 2023. To view the most recent and complete version of the guideline, go online to NCCN.org. NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way.
- Gilreath JA et al. Clin Pharmacol. 2019;11:77-92.
- Verstovsek S. Eur J Haematol. 2013;90(2):89-98.
- Garcia-Montero AC et al. Blood. 2006;108(7):2366-2372.
- Data on file. Blueprint Medicines Corporation, Cambridge, MA. 2021.
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INDICATION
AYVAKIT® (avapritinib) is indicated for the treatment of adult patients with Advanced SM (AdvSM) including patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL).
Limitations of Use: AYVAKIT is not recommended for the treatment of patients with AdvSM with platelet counts of <50 x 109/L.
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