Volume 71, Issue 11 pp. 1585-1593

Original Article

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

B. van Anrooij,

B. van Anrooij

Department of Allergology, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands

University of Groningen, University Medical Center Groningen, GRIAC Research Institute, Groningen, The Netherlands

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J. C. Kluin-Nelemans,

J. C. Kluin-Nelemans

Department of Hematology, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands

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M. Safy,

M. Safy

Department of Allergology, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands

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B. M. J. Flokstra-de Blok,

B. M. J. Flokstra-de Blok

University of Groningen, University Medical Center Groningen, GRIAC Research Institute, Groningen, The Netherlands

Department of General Practice, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands

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J. N. G. Oude Elberink,

Corresponding Author

J. N. G. Oude Elberink

[email protected]

Department of Allergology, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands

University of Groningen, University Medical Center Groningen, GRIAC Research Institute, Groningen, The Netherlands

Correspondence

Joanne N. G. Oude Elberink, MD, PhD, Department of Allergology, Huispostcode AA23, University Medical Center Groningen, P.O.Box 30.001, NL-9700 RB Groningen, The Netherlands.

Tel.: +31 50 3616161

Fax: +31 50 3619308

E-mail: [email protected]

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B. van Anrooij,

B. van Anrooij

Department of Allergology, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands

University of Groningen, University Medical Center Groningen, GRIAC Research Institute, Groningen, The Netherlands

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J. C. Kluin-Nelemans,

J. C. Kluin-Nelemans

Department of Hematology, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands

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M. Safy,

M. Safy

Department of Allergology, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands

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B. M. J. Flokstra-de Blok,

B. M. J. Flokstra-de Blok

University of Groningen, University Medical Center Groningen, GRIAC Research Institute, Groningen, The Netherlands

Department of General Practice, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands

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J. N. G. Oude Elberink,

Corresponding Author

J. N. G. Oude Elberink

[email protected]

Department of Allergology, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands

University of Groningen, University Medical Center Groningen, GRIAC Research Institute, Groningen, The Netherlands

Correspondence

Joanne N. G. Oude Elberink, MD, PhD, Department of Allergology, Huispostcode AA23, University Medical Center Groningen, P.O.Box 30.001, NL-9700 RB Groningen, The Netherlands.

Tel.: +31 50 3616161

Fax: +31 50 3619308

E-mail: [email protected]

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First published: 19 April 2016

https://doi.org/10.1111/all.12920

Citations: 45

Edited by: Werner Aberer

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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.

Supporting Information

References

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Citing Literature

Volume71, Issue11

November 2016

Pages 1585-1593

Filename	Description
all12920-sup-0001-FigS1-TableS2-S3.docxWord document, 76.6 KB	Figure S1 Steps used during the domain identification process for the final mastocytosis quality-of-life questionnaire. Table S2 The 32 MQLQ items selected from the general mastocytosis population Table S3 Characteristics of patients used in the item generation and reduction phase of the MSAF and MQLQ
all12920-sup-0002-TableS1.docxWord document, 44.9 KB	Table S1 all 228 item generated during the item generation phase
all12920-sup-0003-TableS4.docxWord document, 15.6 KB	Table S4 Clinical and biochemical characteristics of responders and non-responders for the cross-sectional validation phase
all12920-sup-0004-SupinfoS1.docxWord document, 37.3 KB	Appendix S1 The Mastocytosis Quality of Life Questionnaire (MQLQ)
all12920-sup-0005-SupinfoS2.docxWord document, 43.3 KB	Appendix S2 Mastocytosis Symptoms Assessment Form (MSAF)

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

Abstract

Background

Methods

Results

Conclusions

Supporting Information

References

Citing Literature

References

Information

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

Abstract

Background

Methods

Results

Conclusions

Supporting Information

References

Citing Literature

References

Related

Information