Association Between Myasthenia Gravis–Activities of Daily Living (MG-ADL) and EQ-5D-5L Utility Values: The Additional Effect of Efgartigimod on Utilities

Sarah Dewilde; Cynthia Z. Qi; Glenn Phillips; Sergio Iannazzo; Mathieu F. Janssen

doi:10.1007/s12325-023-02437-w

Association Between Myasthenia Gravis–Activities of Daily Living (MG-ADL) and EQ-5D-5L Utility Values: The Additional Effect of Efgartigimod on Utilities

Sarah Dewilde
, Cynthia Z. Qi^*
, Glenn Phillips
, Sergio Iannazzo
, Mathieu F. Janssen

^*Corresponding author for this work

Psychiatry

Services in Health Economics
argenx US Inc
argenx Switzerland SA

Research output: Contribution to journal › Article › Academic › peer-review

9 Citations (Scopus)

149 Downloads (Pure)

Abstract

Introduction: For patients with generalized myasthenia gravis (gMG), the association between symptom severity, often measured with the Myasthenia Gravis Activities of Daily Living (MG-ADL) instrument, and utility values is unknown. Methods: Data was analyzed from the phase 3 ADAPT trial, which included adult patients with gMG randomly assigned to treatment with efgartigimod + conventional therapy (EFG + CT) or placebo + CT (PBO + CT). MG-ADL total symptom scores and the EQ-5D-5L, a measure of health-related quality of life (HRQoL), were collected biweekly up to 26 weeks. Utility values were derived from the EQ-5D-5L data with the United Kingdom value set. Descriptive statistics were reported for MG-ADL and EQ-5D-5L at baseline and follow-up. A normal identity-link regression model estimated the association between utility and the eight MG-ADL items. A generalized estimating equations (GEE) model was estimated to predict utility based on the patient’s MG-ADL score and treatment received. Results: A total of 167 patients (84 EFG + CT, 83 PBO + CT) contributed 167 baseline and 2867 follow-up measurements of MG-ADL and EQ-5D-5L. EFG + CT-treated patients experienced more improvements than PBO + CT-treated patients in most MG-ADL items and EQ-5D-5L dimensions, with the largest improvements observed in chewing, brushing teeth/combing hair, eyelid droop (MG-ADL); self-care, usual activities, mobility (EQ-5D-5L). The regression model indicated that individual MG-ADL items contributed differently to utility values, with the largest impact from brushing teeth/combing hair, rising from a chair, chewing, and breathing. The GEE model showed that each unit improvement in MG-ADL led to a statistically significant utility increase of 0.0233 (p < 0.001). In addition, a statistically significant improvement of 0.0598 (p = 0.0079) in utility was found for patients in the EFG + CT group compared to the PBO + CT group. Conclusion: Among patients with gMG, improvements in MG-ADL were significantly associated with higher utility values. MG-ADL scores alone were not sufficient to capture the utility gained from efgartigimod therapy.

Original language	English
Pages (from-to)	1818-1829
Number of pages	12
Journal	Advances in Therapy
Volume	40
Issue number	4
Early online date	Apr 2023
DOIs	https://doi.org/10.1007/s12325-023-02437-w
Publication status	Published - Apr 2023

Bibliographical note

Funding Information:
This study, along with the journal’s rapid service fee, was funded by argenx. The study sponsor was involved in study design, data interpretation, and data and manuscript review. Cynthia Qi, Glenn Phillips, and Sergio Iannazzo are argenx employees. Sarah Dewilde and Mathieu Janssen received funding from argenx to conduct the study. Editorial assistance in the preparation of this article was provided by Meg Franklin, PharmD, PhD, and Samantha Wronski Holloway, MS of Franklin Pharmaceutical Consulting. Support for this assistance was funded by argenx Inc. Sarah Dewilde contributed to the research conceptualization, funding acquisition, methodology, data analysis, and authoring of the manuscript. Mathieu Janssen contributed to the methodology, data analysis, and interpretation. Cynthia Qi, Sergio Iannazzo, and Glenn Phillips contributed to the study conception and design, data analysis, and interpretation. All authors contributed to the development of the manuscript and maintained control over the final content. Sarah Dewilde and Mathieu Janssen received funding from argenx to conduct this analysis and develop this manuscript. Cynthia Qi, Glenn Phillips and Sergio Iannazzo are employees of argenx and own stock/options. This was a secondary analysis of existing data from the ADAPT study. Independent ethics committees and international review boards provided written approval for the ADAPT study protocol and all amendments [21]. The trial was conducted in accordance with Declaration of Helsinki principles [21]. Patients consented both to participation in original ADAPT study and for their data to be published. The datasets generated during and/or analyzed during the current study are not publicly available as they are commercial in confidence.

Publisher Copyright:
© 2023, The Author(s).

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Cite this

@article{3066bfd86a40499da62e4d2da3066469,

title = "Association Between Myasthenia Gravis–Activities of Daily Living (MG-ADL) and EQ-5D-5L Utility Values: The Additional Effect of Efgartigimod on Utilities",

abstract = "Introduction: For patients with generalized myasthenia gravis (gMG), the association between symptom severity, often measured with the Myasthenia Gravis Activities of Daily Living (MG-ADL) instrument, and utility values is unknown. Methods: Data was analyzed from the phase 3 ADAPT trial, which included adult patients with gMG randomly assigned to treatment with efgartigimod + conventional therapy (EFG + CT) or placebo + CT (PBO + CT). MG-ADL total symptom scores and the EQ-5D-5L, a measure of health-related quality of life (HRQoL), were collected biweekly up to 26 weeks. Utility values were derived from the EQ-5D-5L data with the United Kingdom value set. Descriptive statistics were reported for MG-ADL and EQ-5D-5L at baseline and follow-up. A normal identity-link regression model estimated the association between utility and the eight MG-ADL items. A generalized estimating equations (GEE) model was estimated to predict utility based on the patient{\textquoteright}s MG-ADL score and treatment received. Results: A total of 167 patients (84 EFG + CT, 83 PBO + CT) contributed 167 baseline and 2867 follow-up measurements of MG-ADL and EQ-5D-5L. EFG + CT-treated patients experienced more improvements than PBO + CT-treated patients in most MG-ADL items and EQ-5D-5L dimensions, with the largest improvements observed in chewing, brushing teeth/combing hair, eyelid droop (MG-ADL); self-care, usual activities, mobility (EQ-5D-5L). The regression model indicated that individual MG-ADL items contributed differently to utility values, with the largest impact from brushing teeth/combing hair, rising from a chair, chewing, and breathing. The GEE model showed that each unit improvement in MG-ADL led to a statistically significant utility increase of 0.0233 (p < 0.001). In addition, a statistically significant improvement of 0.0598 (p = 0.0079) in utility was found for patients in the EFG + CT group compared to the PBO + CT group. Conclusion: Among patients with gMG, improvements in MG-ADL were significantly associated with higher utility values. MG-ADL scores alone were not sufficient to capture the utility gained from efgartigimod therapy.",

author = "Sarah Dewilde and Qi, \{Cynthia Z.\} and Glenn Phillips and Sergio Iannazzo and Janssen, \{Mathieu F.\}",

note = "Funding Information: This study, along with the journal{\textquoteright}s rapid service fee, was funded by argenx. The study sponsor was involved in study design, data interpretation, and data and manuscript review. Cynthia Qi, Glenn Phillips, and Sergio Iannazzo are argenx employees. Sarah Dewilde and Mathieu Janssen received funding from argenx to conduct the study. Editorial assistance in the preparation of this article was provided by Meg Franklin, PharmD, PhD, and Samantha Wronski Holloway, MS of Franklin Pharmaceutical Consulting. Support for this assistance was funded by argenx Inc. Sarah Dewilde contributed to the research conceptualization, funding acquisition, methodology, data analysis, and authoring of the manuscript. Mathieu Janssen contributed to the methodology, data analysis, and interpretation. Cynthia Qi, Sergio Iannazzo, and Glenn Phillips contributed to the study conception and design, data analysis, and interpretation. All authors contributed to the development of the manuscript and maintained control over the final content. Sarah Dewilde and Mathieu Janssen received funding from argenx to conduct this analysis and develop this manuscript. Cynthia Qi, Glenn Phillips and Sergio Iannazzo are employees of argenx and own stock/options. This was a secondary analysis of existing data from the ADAPT study. Independent ethics committees and international review boards provided written approval for the ADAPT study protocol and all amendments [21]. The trial was conducted in accordance with Declaration of Helsinki principles [21]. Patients consented both to participation in original ADAPT study and for their data to be published. The datasets generated during and/or analyzed during the current study are not publicly available as they are commercial in confidence. Publisher Copyright: {\textcopyright} 2023, The Author(s).",

year = "2023",

month = apr,

doi = "10.1007/s12325-023-02437-w",

language = "English",

volume = "40",

pages = "1818--1829",

journal = "Advances in Therapy",

issn = "0741-238X",

publisher = "Adis",

number = "4",

}

TY - JOUR

T1 - Association Between Myasthenia Gravis–Activities of Daily Living (MG-ADL) and EQ-5D-5L Utility Values

T2 - The Additional Effect of Efgartigimod on Utilities

AU - Dewilde, Sarah

AU - Qi, Cynthia Z.

AU - Phillips, Glenn

AU - Iannazzo, Sergio

AU - Janssen, Mathieu F.

N1 - Funding Information: This study, along with the journal’s rapid service fee, was funded by argenx. The study sponsor was involved in study design, data interpretation, and data and manuscript review. Cynthia Qi, Glenn Phillips, and Sergio Iannazzo are argenx employees. Sarah Dewilde and Mathieu Janssen received funding from argenx to conduct the study. Editorial assistance in the preparation of this article was provided by Meg Franklin, PharmD, PhD, and Samantha Wronski Holloway, MS of Franklin Pharmaceutical Consulting. Support for this assistance was funded by argenx Inc. Sarah Dewilde contributed to the research conceptualization, funding acquisition, methodology, data analysis, and authoring of the manuscript. Mathieu Janssen contributed to the methodology, data analysis, and interpretation. Cynthia Qi, Sergio Iannazzo, and Glenn Phillips contributed to the study conception and design, data analysis, and interpretation. All authors contributed to the development of the manuscript and maintained control over the final content. Sarah Dewilde and Mathieu Janssen received funding from argenx to conduct this analysis and develop this manuscript. Cynthia Qi, Glenn Phillips and Sergio Iannazzo are employees of argenx and own stock/options. This was a secondary analysis of existing data from the ADAPT study. Independent ethics committees and international review boards provided written approval for the ADAPT study protocol and all amendments [21]. The trial was conducted in accordance with Declaration of Helsinki principles [21]. Patients consented both to participation in original ADAPT study and for their data to be published. The datasets generated during and/or analyzed during the current study are not publicly available as they are commercial in confidence. Publisher Copyright: © 2023, The Author(s).

PY - 2023/4

Y1 - 2023/4

N2 - Introduction: For patients with generalized myasthenia gravis (gMG), the association between symptom severity, often measured with the Myasthenia Gravis Activities of Daily Living (MG-ADL) instrument, and utility values is unknown. Methods: Data was analyzed from the phase 3 ADAPT trial, which included adult patients with gMG randomly assigned to treatment with efgartigimod + conventional therapy (EFG + CT) or placebo + CT (PBO + CT). MG-ADL total symptom scores and the EQ-5D-5L, a measure of health-related quality of life (HRQoL), were collected biweekly up to 26 weeks. Utility values were derived from the EQ-5D-5L data with the United Kingdom value set. Descriptive statistics were reported for MG-ADL and EQ-5D-5L at baseline and follow-up. A normal identity-link regression model estimated the association between utility and the eight MG-ADL items. A generalized estimating equations (GEE) model was estimated to predict utility based on the patient’s MG-ADL score and treatment received. Results: A total of 167 patients (84 EFG + CT, 83 PBO + CT) contributed 167 baseline and 2867 follow-up measurements of MG-ADL and EQ-5D-5L. EFG + CT-treated patients experienced more improvements than PBO + CT-treated patients in most MG-ADL items and EQ-5D-5L dimensions, with the largest improvements observed in chewing, brushing teeth/combing hair, eyelid droop (MG-ADL); self-care, usual activities, mobility (EQ-5D-5L). The regression model indicated that individual MG-ADL items contributed differently to utility values, with the largest impact from brushing teeth/combing hair, rising from a chair, chewing, and breathing. The GEE model showed that each unit improvement in MG-ADL led to a statistically significant utility increase of 0.0233 (p < 0.001). In addition, a statistically significant improvement of 0.0598 (p = 0.0079) in utility was found for patients in the EFG + CT group compared to the PBO + CT group. Conclusion: Among patients with gMG, improvements in MG-ADL were significantly associated with higher utility values. MG-ADL scores alone were not sufficient to capture the utility gained from efgartigimod therapy.

AB - Introduction: For patients with generalized myasthenia gravis (gMG), the association between symptom severity, often measured with the Myasthenia Gravis Activities of Daily Living (MG-ADL) instrument, and utility values is unknown. Methods: Data was analyzed from the phase 3 ADAPT trial, which included adult patients with gMG randomly assigned to treatment with efgartigimod + conventional therapy (EFG + CT) or placebo + CT (PBO + CT). MG-ADL total symptom scores and the EQ-5D-5L, a measure of health-related quality of life (HRQoL), were collected biweekly up to 26 weeks. Utility values were derived from the EQ-5D-5L data with the United Kingdom value set. Descriptive statistics were reported for MG-ADL and EQ-5D-5L at baseline and follow-up. A normal identity-link regression model estimated the association between utility and the eight MG-ADL items. A generalized estimating equations (GEE) model was estimated to predict utility based on the patient’s MG-ADL score and treatment received. Results: A total of 167 patients (84 EFG + CT, 83 PBO + CT) contributed 167 baseline and 2867 follow-up measurements of MG-ADL and EQ-5D-5L. EFG + CT-treated patients experienced more improvements than PBO + CT-treated patients in most MG-ADL items and EQ-5D-5L dimensions, with the largest improvements observed in chewing, brushing teeth/combing hair, eyelid droop (MG-ADL); self-care, usual activities, mobility (EQ-5D-5L). The regression model indicated that individual MG-ADL items contributed differently to utility values, with the largest impact from brushing teeth/combing hair, rising from a chair, chewing, and breathing. The GEE model showed that each unit improvement in MG-ADL led to a statistically significant utility increase of 0.0233 (p < 0.001). In addition, a statistically significant improvement of 0.0598 (p = 0.0079) in utility was found for patients in the EFG + CT group compared to the PBO + CT group. Conclusion: Among patients with gMG, improvements in MG-ADL were significantly associated with higher utility values. MG-ADL scores alone were not sufficient to capture the utility gained from efgartigimod therapy.

UR - https://www.scopus.com/pages/publications/85149210358

U2 - 10.1007/s12325-023-02437-w

DO - 10.1007/s12325-023-02437-w

M3 - Article

C2 - 36867327

AN - SCOPUS:85149210358

SN - 0741-238X

VL - 40

SP - 1818

EP - 1829

JO - Advances in Therapy

JF - Advances in Therapy

IS - 4

ER -

Association Between Myasthenia Gravis–Activities of Daily Living (MG-ADL) and EQ-5D-5L Utility Values: The Additional Effect of Efgartigimod on Utilities

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