Patient-reported disease-specific quality-of-life and symptom severity in systemic mastocytosis

B van Anrooij; J C Kluin-Nelemans; M Safy; B M J Flokstra-de Blok; J N G Oude Elberink

doi:10.1111/all.12920

Patient-reported disease-specific quality-of-life and symptom severity in systemic mastocytosis

Allergy. 2016 Nov;71(11):1585-1593. doi: 10.1111/all.12920. Epub 2016 Jun 23.

Authors

B van Anrooij^{1

2}, J C Kluin-Nelemans³, M Safy¹, B M J Flokstra-de Blok^{2

4}, J N G Oude Elberink^{5

6}

Affiliations

¹ Department of Allergology, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands.
² University of Groningen, University Medical Center Groningen, GRIAC Research Institute, Groningen, The Netherlands.
³ Department of Hematology, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands.
⁴ Department of General Practice, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands.
⁵ Department of Allergology, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands. j.n.g.oude.elberink@umcg.nl.
⁶ University of Groningen, University Medical Center Groningen, GRIAC Research Institute, Groningen, The Netherlands. j.n.g.oude.elberink@umcg.nl.

PMID: 27089859
DOI: 10.1111/all.12920

Abstract

Background: Presently, no validated data exist on symptom severity and disease-specific quality-of-life (QoL) for patients with mastocytosis. Simultaneously, clinical trials and drug application processes increasingly mandate reporting patients' perspectives on symptoms and QoL. We report on the development and validation of the mastocytosis quality-of-life questionnaire (MQLQ) and the mastocytosis symptom assessment form (MSAF).

Methods: Both outcome measures were developed in a standardized stepwise method, starting with the identification of items in focus groups (n = 12), item reduction and subsequent cross-sectional validation in a 63% female cohort of 164 adult patients with indolent systemic mastocytosis.

Results: The MSAF reveals that fatigue is the severest mastocytosis symptom while the MQLQ indicates that fear of anaphylaxis mostly impacts QoL. Cross-sectional validity was assessed by correlating both individual domains and the total scores of the MQLQ and MSAF with independent measures of mastocytosis. The total scores of both the MQLQ (P < 0.001; Spearman's r: 0.568) and the MSAF (P < 0.001; Spearman's r: 0.559) correlated significantly with the consensus on physician-scored mediator symptoms. The MQLQ domains displayed a high internal consistency (Cronbach's alpha: 0.841-0.958) and the domains 'bones', 'skin symptoms' and 'anaphylaxis' differed significantly between patients with and without osteoporosis, urticaria pigmentosa or anaphylaxis, respectively (P < 0.001).

Conclusions: The MQLQ is the first disease-specific QoL questionnaire for mastocytosis and is complemented by the MSAF, a short and convenient symptom scoring form. Both patient-reported outcome measures are valid, reliable and discriminate between patients with different disease characteristics, making them useful instruments for clinical research.

Keywords: mastocytosis; mastocytosis quality-of-life questionnaire; mastocytosis symptom assessment form; quality-of-life; systemic mastocytosis.

MeSH terms

Cross-Sectional Studies
Female
Humans
Male
Mastocytosis, Systemic / diagnosis*
Mastocytosis, Systemic / epidemiology*
Quality of Life*
Self Report
Severity of Illness Index
Surveys and Questionnaires
Symptom Assessment