Introduction
Methods
Targeted literature review
Expert advice meetings (i.e., interviews with clinical therapeutic area experts)
Concept elicitation interviews
Study overview
Interview conduct
Qualitative data management and coding
Concept selection and questionnaire construction for the Advanced Systemic Mastocytosis Symptom Assessment Form (AdvSM-SAF) and Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF)
Cognitive debriefing interviews for the AdvSM-SAF and ISM-SAF
Study overview
Interview conduct
Data management and analysis
Results
Literature review
Expert advice meetings (interviews with clinical therapeutic area experts)
Advanced systemic mastocytosis
Literature review
Expert advice meetings with AdvSM clinical therapeutic area experts
Concept elicitation interviews
AdvSM patients | ISM patients | |||
---|---|---|---|---|
CE interviews Total (N = 12) n (%)* | CD interviews Total (N = 13) n (%)* | CE interviews Total (N = 15)† n (%)* | CD interviews Total (N = 10) n (%)* | |
Age (years) | ||||
Range | 38.9–77.5 | 49.8–82.6 | 26.7–69.0 | 29.4–56.7 |
Mean (standard deviation [SD]) | 62.1 (12.9) | 68.7 (10.9) | 48.3 (14.2) | 45.0 (10.0) |
Gender | ||||
Female | 7 (58.3%) | 7 (53.8%) | 14 (93.3%) | 8 (80.0%) |
Male | 5 (41.7%) | 6 (46.2%) | 1 (6.7%) | 2 (20.0%) |
Race | ||||
White | 7 (58.3%) | 12 (92.3%) | 14 (93.3%) | 10 (100.0%) |
Not answered | 4 (33.3%) | 0 (0.0%) | – | – |
Asian | 1 (8.3%) | 0 (0.0%) | – | – |
American Indian or Alaska Native | 0 (0.0%) | 1 (7.7%) | 1 (6.7%) | 0 (0.0%) |
Ethnicity | ||||
Data not collected in Germany | 7 (58.3%) | 7 (53.8%) | – | – |
No, not Spanish/Hispanic/Latino | 4 (33.3%) | 5 (38.5%) | 13 (86.7%) | 10 (100.0%) |
Yes, Puerto Rican | – | – | 1 (6.7%) | 0 (0.0%) |
Not answered | 1 (8.3%) | 1 (7.7%) | 1 (6.7%) | 0 (0.0%) |
Highest level of education | ||||
High school diploma (or GED) or less | 3 (25.0%) | 1 (7.7%) | 2 (13.3%) | 2 (20.0%) |
Some college or certificate program | 4 (33.3%) | 7 (53.8%) | 3 (20.0%) | 2 (20.0%) |
College or university degree (two- or four-year) | 2 (16.7%) | 4 (30.8%) | 8 (53.3%) | 3 (30.0%) |
Graduate degree | 3 (25.0%) | 1 (7.7%) | 1 (6.7%) | 3 (30.0%) |
Other | – | – | 1 (6.7%) | 0 (0.0%) |
Work status | ||||
Retired | 6 (50.0%) | 9 (69.2%) | 1 (6.7%) | 0 (0.0%) |
On disability | 3 (25.0%) | 2 (15.4%) | 7 (46.7%) | 3 (30.0%) |
Working part-time | 3 (25.0%) | 2 (15.4%) | 3 (20.0%) | 2 (20.0%) |
Working full-time | 1 (8.3%) | 1 (7.7%) | 3 (20.0%) | 4 (40.0%) |
Homemaker | – | – | 2 (13.3%) | 0 (0.0%) |
Student | – | – | 1 (6.7%) | 0 (0.0%) |
Unemployed | – | – | 0 (0.0%) | 1 (10.0%) |
Type of systemic mastocytosis | ||||
AHN | 6 (50.0%) | 8 (61.5%) | – | – |
ASM | 3 (25.0%) | 2 (15.4%) | – | – |
MCL | 3 (25.0%) | 3 (23.1%) | – | – |
ISM | – | – | 13 (81.3%) | 10 (100.0%) |
SSM | – | – | 3 (18.7%) | 0 (0.0%) |
Time since diagnosis (years) | ||||
Range | 0.2–3.8 | 0.1–8.4 | 0.3–18.1 | – |
Mean (SD) | 0.7 (1.0) | 0.7 (2.6) | 4.3 (4.1) | – |
Other health conditions (participant-reported)‡ | ||||
Heart disease | 2 (16.7%) | 0 (0.0%) | 1 (6.7%) | 0 (0.0%) |
High blood pressure | 2 (16.7%) | 4 (30.8%) | 2 (13.3%) | 2 (20.0%) |
Chronic obstructive pulmonary disease (COPD) | 0 (0.0%) | 3 (23.1%) | 0 (0.0%) | 1 (10.0%) |
High cholesterol | 2 (16.7%) | 0 (0.0%) | 2 (13.3%) | 0 (0.0%) |
Asthma | – | – | 2 (13.3%) | 1 (10.0%) |
Fibromyalgia | – | – | 2 (13.3%) | 2 (20.0%) |
Liver disease | 2 (16.7%) | 1 (7.7%) | 1 (6.7%) | 0 (0.0%) |
Kidney disorder | – | – | 1 (6.7%) | 0 (0.0%) |
Thyroid disease | 2 (16.7%) | 2 (15.4%) | 3 (20.0%) | 1 (10.0%) |
Depression/anxiety | 1 (8.3%) | 2 (15.4%) | 8 (53.3%) | 7 (70.0%) |
Migraine headaches | – | – | 4 (26.7%) | 0 (0.0%) |
Cancer | 1 (8.3%) | 1 (7.7%) | 0 (0.0%) | 2 (20.0%) |
Stomach/intestinal disorder | 1 (8.3%) | 1 (7.7%) | 3 (20.0%) | 4 (40.0%) |
None | 2 (16.7%) | 2 (15.4%) | 3 (20.0%) | 0 (0.0%) |
Other | 2 (16.7%) | 3 (23.1%) | 5 (33.3%) | 3 (30.0%) |
Not answered | 1 (8.3%) | 1 (7.7%) | 0 (0.0%) | 1 (10.0%) |
Treatments (current and past)‡;§ | ||||
Tyrosine kinase inhibitors | Current: 6 (50.0%) Past: 3 (25.0%) | Current: 6 (46.2%) Historical: 3 (23.1%) | Current: 3 (18.8%) Past: 1 (6.3%) | Current: 1 (10.0%) |
H1 antagonists | Current: 5 (41.7%) Past: 2 (16.7%) | Current: 4 (30.8%) Historical: 3 (23.1%) | Current: 16 (100.0%) Past: 3 (18.8%) | Current: 7 (70.0%) Past: 2 (20.0%) |
Corticosteroids | Current: 5 (41.7%) Past: 1 (8.3%) | Current: 3 (23.1%) Historical: 2 (15.4%) | Current: 2 (12.5%) Past: 4 (25.0%) | Current: 1 (10.0%) Past: 6 (60.0%) |
Proton pump inhibitor | Current: 5 (41.7%) | Current: 5 (38.5%) | Current: 3 (18.8%) Past: 1 (6.3%) | Current: 2 (20.0%) Past: 1 (10.0%) |
H2 antagonists | Current: 4 (33.3%) Past: 1 (8.3%) | Current: 5 (38.5%) Historical: 1 (7.7%) | Current: 14 (87.5%) Past: 0 (0.0%) | Current: 6 (60.0%) Past: 2 (20.0%) |
Non-steroidal anti-inflammatory drugs | Current: 4 (33.3%) | Current: 4 (30.8%) | Current: 3 (18.8%) Past: 0 (0.0%) | Current: 3 (30.0%) Past: 1 (10.0%) |
Cytokine/immunomodulatory drugs | Current: 2 (16.7%) Past: 1 (8.3%) | Current: 1 (7.7%) Historical: 1 (7.7%) | Current: 2 (12.5%) Past: 3 (18.8%) | Past: 2 (20.0%) |
Beta-adrenergic agonists | Current: 1 (8.3%) | – | Current: 3 (18.8%) Past: 0 (0.0%) | Current: 3 (30.0%) Past: 1 (10.0%) |
Anti-IgE | – | – | Current: 0 (0.0%) Past: 1 (6.3%) | – |
Cannabis | Current: 1 (8.3%) | – | – | – |
Aldactone® | Current: 1 (8.3%) | – | – | – |
Zofran® | Current: 1 (8.3%) | – | – | – |
Tramadol | Current: 1 (8.3%) | – | – | – |
Zaditor® | Current: 1 (8.3%) | – | – | – |
Hydroxycarbamide | Current: 1 (8.3%) | – | – | – |
Zoledronate | Current: 1 (8.3%) | – | – | – |
Purine nucleoside analogues | Current: 0 (0.0%) Past: 2 (16.7%) | Historical: 3 (23.1%) | Current: 0 (0.0%) Past: 1 (6.3%) | – |
Leukotriene antagonist | – | Current: 1 (7.7%) | Current: 7 (43.8%) Past: 0 (0.0%) | Current: 3 (30.0%) |
Other | – | Current: 6 (46.2%) | Current: 2 (12.5%) Past: 4 (25.0%) | Current: 8 (80.0%) Past: 2 (20.0%) |
Questionnaire construction
Cognitive debriefing of the advanced systemic mastocytosis symptom assessment form (AdvSM-SAF)
Concept | Domain | General concept |
---|---|---|
Abdominal pain | Gastrointestinal symptom severity | Total symptom severity |
Nausea | ||
Vomiting | ||
Diarrhea | ||
Spots | Skin symptom severity | |
Itching | ||
Flushing | ||
Fatigue | Fatigue severity | |
Vomiting frequency | Vomiting frequency | Vomiting frequency |
Diarrhea frequency | Diarrhea frequency | Diarrhea frequency |
Indolent systemic mastocytosis and smoldering systemic mastocytosis
Literature review
Expert advice meetings with ISM and SSM clinical therapeutic area experts
Concept elicitation interviews
Questionnaire construction
Cognitive debriefing of the Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF)
Concept | Domain | General concept |
---|---|---|
Abdominal pain | Gastrointestinal symptom severity | Total symptom severity |
Nausea | ||
Diarrhea | ||
Spots | Skin symptom severity | |
Itching | ||
Flushing | ||
Bone pain | Bone pain | |
Fatigue | Fatigue | |
Dizziness | Dizziness | |
Brain fog | Brain fog | |
Headache | Headache | |
Diarrhea frequency | Diarrhea frequency | Diarrhea frequency |