Appointment sheet
Indolent and smouldering systemic mastocytosis
Prepared with OnCo (onco.cc/prep/indolent-systemic-mastocytosis/). Orientation, not medical advice; your team knows your case.
My details
Name
Date of appointment
Hospital and clinician
Who is coming with me
What I know, what is unclear, changes to discuss
Saved in this browserWhat I know so far
What is unclear to me
Changes since last time
My questions
18 on the sheetNewly diagnosed
- 1.What is my exact diagnosis, stage, and grade, and which tests established them?
- 2.Which biomarkers have been tested on my tumour (for example Serum tryptase, KIT D816V by high-sensitivity PCR in blood, Marrow mast cell aggregates with CD25, CD2 and CD30 expression, B findingsdefining smouldering disease, SRSF2, ASXL1 and RUNX1 mutations), and what were the results?
- 3.Which subtype is my cancer, and does that change the recommended treatment?
- 4.Is germline (inherited) genetic testing recommended for me or my family?
Diagnosis
- 5.For my situation (diagnosis), which of the standard options do you recommend and why?
Symptom control
- 6.For my situation (symptom control), which of the standard options do you recommend and why?
Moderate to severe symptoms despite supportive care
- 7.For my situation (moderate to severe symptoms despite supportive care), which of the standard options do you recommend and why?
- 8.Am I a candidate for Avapritinib, Cladribine, Interferon alfa-2a/2b, and what side effects should I expect?
- 9.How do the results of (PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo apply to someone like me?
Trials
- 10.For my situation (trials), which of the standard options do you recommend and why?
- 11.Am I a candidate for Elenestinib, Bezuclastinib, and what side effects should I expect?
- 12.How do the results of (HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis and (Summit) A Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients With Indolent or Smoldering Systemic Mastocytosis apply to someone like me?
Monitoring
- 13.For my situation (monitoring), which of the standard options do you recommend and why?
Any stage
- 14.Are there clinical trials I could join, for example of Avapritinib, Elenestinib, Bezuclastinib, (HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis?
- 15.Would a second opinion at a high-volume centre change anything, and can you help arrange it?
- 16.What supportive care (symptom control, nutrition, exercise, mental health, financial help) is available from the start?
- 17.I read that “Whether avapritinib alters the long-term course or only symptoms is unknown”. How does that affect my plan?
- 18.I read that “Anaphylaxis remains life-threatening and unpredictable”. How does that affect my plan?
The words I may hear
- Molecular response (MMR, MR4, treatment-free remission): In chronic myeloid leukaemia, how far the leukaemia gene signal in the blood has fallen, measured in logs: a 1,000-fold drop is a major molecular response, a 10,000-fold drop (MR4) is 'deep'.
- Driver mutation: One of the few mutations in a tumour that actually causes it to grow.
Tests and results to bring
Diagnosis: Serum tryptase, KIT D816V testing in peripheral blood, bone marrow biopsy with flow cytometry, tryptase gene copy number, bone density scan and screening for B and C findings.
Biomarker results to ask for: Serum tryptase (adjusted for hereditary alpha-tryptasaemia copy number), KIT D816V by high-sensitivity PCR in blood (allele burden tracks response), Marrow mast cell aggregates with CD25, CD2 and CD30 expression, B findings (marrow burden over 30 percent, tryptase over 200 ng/mL, organomegaly) defining smouldering disease, SRSF2, ASXL1 and RUNX1 mutations (progression risk), Bone density scan (osteoporosis).
Scans and tests linked to this cancer: Histopathology & immunohistochemistry, Liquid biopsy (ctDNA), Multiparameter flow cytometry MRD.
Bring copies of scan reports, pathology and blood results, and a list of every medicine and supplement.
The treatments I may be offered
- Symptom control: H1 and H2 antihistamines, cromoglicate, leukotriene antagonists, proton-pump inhibitors; omalizumab for recurrent anaphylaxis; adrenaline autoinjectors for all; venom immunotherapy after sting anaphylaxis; bisphosphonates for osteoporosis. (Electronic patient-reported outcome (ePRO) symptom monitoring)
- Trials: Elenestinib (HARBOR) and bezuclastinib (Summit) as more selective KIT D816V inhibitors. (Elenestinib, (HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis, Bezuclastinib, (Summit) A Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients With Indolent or Smoldering Systemic Mastocytosis)
- Monitoring: Tryptase, KIT allele burden and blood count yearly; bone density; marrow re-examination only if progression is suspected. (Liquid biopsy (ctDNA), Molecular response (MMR, MR4, treatment-free remission))
- Moderate to severe symptoms despite supportive care: Avapritinib 25 mg daily (PIONEER, approved 2023), avoided when platelets are below 50 x 10^9/L; cladribine or interferon alfa as older cytoreductive options. (Avapritinib, (PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo , Cladribine, Interferon alfa-2a/2b, Small-molecule kinase inhibitors)
From the standard of care recorded for this cancer; which apply depends on your stage and biomarkers. Ask which the team recommends and why.
Answers and next steps
Saved in this browserWhat I was told
Agreed next steps, dates and who to call