Patient profiles are fictional portrayals based on actual clinical trial experiences with AYVAKYT
Select each of the cases below to learn more
Leon
68-year-old male diagnosed with SM-AHN
Celine
65-year-old female diagnosed with ASM
Parminder
67-year-old male diagnosed with SM-AHN
Adrienne
64-year-old female diagnosed with MCL
Leon
68-year-old male diagnosed with SM-AHN
Patient history:
Leon was diagnosed with SM-AHN (low-risk MDS) 3 months ago
Initiated treatment with a tyrosine kinase inhibitor but discontinued due to tolerability issues
Baseline platelet count was 65 x 109/L
Introduction of AYVAKYT:
The patient initiated AYVAKYT 200 mg once daily, and platelet counts were monitored every 2 weeks for the first 8 weeks as per dosing guidelines1
Platelet count decreased to 45 x 109/L after 4 weeks of treatment
Treatment was interrupted until platelet count recovered to >50 x 109/L, due to increased risk of ICH, as per dosing guidelines1
Treatment evolution:
After 2 weeks, the platelet count did not improve, and the physician ordered a transfusion
Platelet count improved to >50 x 109/L, and treatment was resumed at a reduced dose of 100 mg once daily with monitoring1
After 6 months of treatment, repeated bone marrow biopsy showed a significant reduction in bone marrow mast cell aggregates, and a steady improvement in symptoms was reported
MDS=myelodysplastic syndrome
Celine
65-year-old female diagnosed with ASM
Patient history:
Celine was diagnosed with ASM
Initiated treatment with interferon alfa
The treating physician decided to switch therapies due to unstable serum tryptase levels and tolerability issues
Introduction of AYVAKYT:
The patient initiated AYVAKYT 200 mg once daily and platelet counts were monitored every 2 weeks for the first 8 weeks, as per dosing guidelines1
Platelet count at initiation was 188 x 109/L
Mild (Grade 1) facial oedema and peripheral oedema were observed within a week. Platelet count was 140 x 109/L with no other cytopenias after 2 weeks of treatment
The patient started to experience cognitive effects such as losing her keys and word-finding difficulty, so treatment was interrupted, as per dosing guidelines1
Treatment evolution:
After 2 weeks, the cognitive effects improved, and treatment was resumed at a reduced dose of 100 mg once daily with monitoring1
After 1 year of treatment, the patient remained on AYVAKYT therapy at 100 mg once daily and was tolerating it well
MDS=myelodysplastic syndrome
Parminder
67-year-old male diagnosed with SM-AHN
Patient history:
Parminder presented with fatigue, mild ascites and maculopapular rash with 15% monocytes in circulating blood
Initiated treatment with azacitidine (x4 cycles) but no improvement in symptoms was observed
Re-evaluation confirmed a revised diagnosis of SM-AHN
Introduction of AYVAKYT:
The patient initiated AYVAKYT 200 mg once daily, and platelet counts were monitored every 2 weeks for the first 8 weeks, as per dosing guidelines1
Worsening peripheral oedema (Grade 3) was observed after 3 months of treatment
Treatment was interrupted until resolution to baseline, as per dosing guidelines1
Treatment evolution:
After 1 week, oedema resolved and treatment was resumed at a reduced dose of 100 mg once daily with monitoring1
After 2 years of treatment, the patient remained on AYVAKYT therapy at 100 mg once daily and was tolerating it well
MDS=myelodysplastic syndrome
Adrienne
64-year-old female diagnosed with MCL
Patient history:
Adrienne was diagnosed with MCL
Initiated treatment with cladribine (x2 cycles) to cytoreduce abnormally high mast cell counts. In parallel, an allogenic consult was initiated
Symptoms improved but the patient was ruled out for bone marrow transplant due to the lack of available donors
Additional treatment options were discussed with the patient
Introduction of AYVAKYT:
The patient initiated AYVAKYT 200 mg once daily, and platelet counts were monitored every 2 weeks for the first 8 weeks as per dosing guidelines1
Platelet count at initiation was 95 x 109/L
After 6 weeks of treatment, 5% of bone marrow mast cells were detected. Thrombocytopenia improved to Grade 1 (160 x 109/L)
Grade 2 anaemia and Grade 2 periorbital oedema were observed, so treatment was interrupted for 1 week
Treatment evolution:
After 1 week, adverse reactions improved, and treatment was resumed at a reduced dose of 100 mg once daily with monitoring1
After 1 year of treatment, repeated bone marrow biopsy showed an improvement in mast cell measures
MDS=myelodysplastic syndrome
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.
References
AYVAKYT® (avapritinib). Summary of Product Characteristics. Available at: https://www.ema.europa.eu/en/documents/product-information/ayvakyt-epar-product-information_en.pdf. Accessed February 2024.
The information on this website is intended for an international healthcare professional audience outside the United States and United Kingdom only
I confirm that I am a healthcare professional
I confirm that I am outside the US and UK
Our use of cookies
We use cookies to make our site work. We'd also like to set optional analytics cookies to help us improve it. We won't set optional cookies unless you enable them. Using this tool will set a cookie on your device to remember your preferences.
For more detailed information about the cookies we use, see our Cookie Policy.
Necessary cookies
Necessary cookies enable core functionality such as security, network management and accessibility. You may disable these by changing your browser settings, but this may affect how the website functions.
Analytics cookies
We'd like to set Google Analytics cookies to help us to improve our website by collecting and reporting information on how you use it. For more information on how these cookies work, please see our Cookie Policy. The cookies collect information in an anonymous form.
We use cookies that are strictly necessary to operate this website. We also use optional cookies to improve your accessibility to our website and its functionality, to analyse and track your use of our website, and to give you the best possible user experience. By clicking on the “Reject All Cookies” button, you understand that strictly necessary cookies will continue to be deployed for the operation of this website. For more detailed information on our use of cookies on this website, please read our Cookie Policy, which also explains how to customise your cookies settings. You can also “Accept All Cookies” or customise your cookies settings by clicking on the buttons provided below. Click “Cookie Settings” to update your personal cookie preferences or review our Privacy Policy.