Psychometric evaluation of the Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF©) and determination of a threshold score for moderate symptoms
Abstract Background The Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF) (©Blueprint Medicines Corporation), a 12-item daily diary that assesses 11 signs and symptoms of indolent systemic mastocytosis (ISM) and smoldering systemic mastocytosis (SSM), was psychometrically evaluated am...
| Main Authors: | , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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BMC
2023-03-01
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| Series: | Orphanet Journal of Rare Diseases |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s13023-023-02661-1 |
| _version_ | 1797859833146245120 |
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| author | Alan L. Shields Fiona Taylor Roger E. Lamoureux Brad Padilla Kas Severson Tanya Green Anthony L. Boral Cem Akin Frank Siebenhaar Brenton Mar |
| author_facet | Alan L. Shields Fiona Taylor Roger E. Lamoureux Brad Padilla Kas Severson Tanya Green Anthony L. Boral Cem Akin Frank Siebenhaar Brenton Mar |
| author_sort | Alan L. Shields |
| collection | DOAJ |
| description | Abstract Background The Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF) (©Blueprint Medicines Corporation), a 12-item daily diary that assesses 11 signs and symptoms of indolent systemic mastocytosis (ISM) and smoldering systemic mastocytosis (SSM), was psychometrically evaluated among patients with ISM. Additionally, thresholds of the ISM-SAF total symptom score (TSS) to distinguish patients with moderate to severe symptoms from those with mild symptoms were evaluated. Methods The ISM-SAF was completed daily as an electronic diary in a prospective, observational study utilizing an online survey of patients with ISM in the United States. Descriptive statistics, psychometric analyses, and analyses to estimate ISM-SAF TSS clinical cutoff values were conducted. Results A total of 103 patients (81.6% female; mean age = 50.2 [± 12.6]) with a self-reported diagnosis of ISM or SSM (58 of whom also had a medically documented diagnosis) contributed to the analyses. Psychometric analysis supported the trustworthiness of the biweekly TSS, which was reliable (α > 0.8, ICC > 0.9), construct-valid, and able to distinguish among clinically distinct groups as specified by the Patient Global Impression of Severity, 12-item Short-Form Health Survey, and Mastocytosis Quality of Life Questionnaire (p < 0.01). A biweekly ISM-SAF TSS from 21 to 28 begins to distinguish the moderately to severely symptomatic ISM/SSM patients from mildly symptomatic patients. Conclusion The biweekly TSS of ISM-SAF was reliable, construct-valid, and able to distinguish among clinically distinct groups. A cut-off value of 28 is a conservative threshold that can be used for screening purposes in future clinical studies to identify patients with at least a moderate severity of ISM symptoms. |
| first_indexed | 2024-04-09T21:36:00Z |
| format | Article |
| id | doaj.art-94b4b613e6f74b538a3b3852443317a6 |
| institution | Directory Open Access Journal |
| issn | 1750-1172 |
| language | English |
| last_indexed | 2024-04-09T21:36:00Z |
| publishDate | 2023-03-01 |
| publisher | BMC |
| record_format | Article |
| series | Orphanet Journal of Rare Diseases |
| spelling | doaj.art-94b4b613e6f74b538a3b3852443317a62023-03-26T11:17:42ZengBMCOrphanet Journal of Rare Diseases1750-11722023-03-0118111110.1186/s13023-023-02661-1Psychometric evaluation of the Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF©) and determination of a threshold score for moderate symptomsAlan L. Shields0Fiona Taylor1Roger E. Lamoureux2Brad Padilla3Kas Severson4Tanya Green5Anthony L. Boral6Cem Akin7Frank Siebenhaar8Brenton Mar9Adelphi ValuesAdelphi ValuesAdelphi ValuesAdelphi ValuesAdelphi ValuesBlueprint MedicinesBlueprint MedicinesUniversity of MichiganInstitute of Allergology, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität Zu Berlin, and Berlin Institute of HealthBlueprint MedicinesAbstract Background The Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF) (©Blueprint Medicines Corporation), a 12-item daily diary that assesses 11 signs and symptoms of indolent systemic mastocytosis (ISM) and smoldering systemic mastocytosis (SSM), was psychometrically evaluated among patients with ISM. Additionally, thresholds of the ISM-SAF total symptom score (TSS) to distinguish patients with moderate to severe symptoms from those with mild symptoms were evaluated. Methods The ISM-SAF was completed daily as an electronic diary in a prospective, observational study utilizing an online survey of patients with ISM in the United States. Descriptive statistics, psychometric analyses, and analyses to estimate ISM-SAF TSS clinical cutoff values were conducted. Results A total of 103 patients (81.6% female; mean age = 50.2 [± 12.6]) with a self-reported diagnosis of ISM or SSM (58 of whom also had a medically documented diagnosis) contributed to the analyses. Psychometric analysis supported the trustworthiness of the biweekly TSS, which was reliable (α > 0.8, ICC > 0.9), construct-valid, and able to distinguish among clinically distinct groups as specified by the Patient Global Impression of Severity, 12-item Short-Form Health Survey, and Mastocytosis Quality of Life Questionnaire (p < 0.01). A biweekly ISM-SAF TSS from 21 to 28 begins to distinguish the moderately to severely symptomatic ISM/SSM patients from mildly symptomatic patients. Conclusion The biweekly TSS of ISM-SAF was reliable, construct-valid, and able to distinguish among clinically distinct groups. A cut-off value of 28 is a conservative threshold that can be used for screening purposes in future clinical studies to identify patients with at least a moderate severity of ISM symptoms.https://doi.org/10.1186/s13023-023-02661-1Psychometric evaluationInstrument developmentPatient-reported outcomesIndolent systemic mastocytosis |
| spellingShingle | Alan L. Shields Fiona Taylor Roger E. Lamoureux Brad Padilla Kas Severson Tanya Green Anthony L. Boral Cem Akin Frank Siebenhaar Brenton Mar Psychometric evaluation of the Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF©) and determination of a threshold score for moderate symptoms Orphanet Journal of Rare Diseases Psychometric evaluation Instrument development Patient-reported outcomes Indolent systemic mastocytosis |
| title | Psychometric evaluation of the Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF©) and determination of a threshold score for moderate symptoms |
| title_full | Psychometric evaluation of the Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF©) and determination of a threshold score for moderate symptoms |
| title_fullStr | Psychometric evaluation of the Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF©) and determination of a threshold score for moderate symptoms |
| title_full_unstemmed | Psychometric evaluation of the Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF©) and determination of a threshold score for moderate symptoms |
| title_short | Psychometric evaluation of the Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF©) and determination of a threshold score for moderate symptoms |
| title_sort | psychometric evaluation of the indolent systemic mastocytosis symptom assessment form ism saf c and determination of a threshold score for moderate symptoms |
| topic | Psychometric evaluation Instrument development Patient-reported outcomes Indolent systemic mastocytosis |
| url | https://doi.org/10.1186/s13023-023-02661-1 |
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