PRO30 ECONOMIC BURDEN OF POMPE DISEASE IN THE RUSSIAN FEDERATION

A. Kolbin, Velum, Y. Balykina, M. Proskurin, O. Karpov

Research outputpeer-review

Abstract

Introduction: Diary-derived symptom score and rescue medication use endpoints, such as symptom-free days (SFDs) and rescue medication-free days (RFD), are frequently used as clinical trial endpoints. Estimates of meaningful change for SFDs and RFDs have not been generated in pediatric populations. This research aimed to generate evidence supporting estimates of the individual within-patient changes that constitute an important or meaningful change in SFDs, RFDs, and updated estimates on the Childhood Asthma Control Test (C-ACT) in pediatric asthma populations aged 5-11 years. Methods: Semistructured, qualitative interviews were conducted with children (ages 8-11 years) who had asthma and parents/caregivers of children (4-11 years) with asthma. Before the interview (4-9 days) participants were asked to complete a morning and evening diary. Results: On average, parent/caregiver estimates of the difference in SFDs between a “very bad” and a “little bad” week for their children's asthma were largely concordant with the values reported by their children (differences of 1.8 and 1.4 SFDs, respectively). Both parents/caregivers and children were able to articulate what a meaningful level of change would be on the C-ACT at the item level. This qualitative study generated C-ACT item-level meaningful change estimates in the region of 1-3 category change, which potentially suggests that, if scaled up to represent C-ACT total score, this would lead to change estimates of 7-15 points. Conclusions: Our findings suggest that both children with asthma and parents/caregivers can quantitatively estimate and to some extent qualitatively articulate meaningful change in SFDs and RFDs.

Original languageEnglish
Pages (from-to)S340
Number of pages1
JournalValue in Health
Volume22
Issue numberSuppl.2
DOIs
Publication statusPublished - 1 Mar 2019

Cite this

Kolbin, A. ; Velum, ; Balykina, Y. ; Proskurin, M. ; Karpov, O. / PRO30 ECONOMIC BURDEN OF POMPE DISEASE IN THE RUSSIAN FEDERATION. In: Value in Health. 2019 ; Vol. 22, No. Suppl.2. pp. S340.
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PRO30 ECONOMIC BURDEN OF POMPE DISEASE IN THE RUSSIAN FEDERATION. / Kolbin, A.; Velum, ; Balykina, Y.; Proskurin, M.; Karpov, O.

In: Value in Health, Vol. 22, No. Suppl.2, 01.03.2019, p. S340.

Research outputpeer-review

TY - JOUR

T1 - PRO30 ECONOMIC BURDEN OF POMPE DISEASE IN THE RUSSIAN FEDERATION

AU - Kolbin, A.

AU - Velum, null

AU - Balykina, Y.

AU - Proskurin, M.

AU - Karpov, O.

PY - 2019/3/1

Y1 - 2019/3/1

N2 - Introduction: Diary-derived symptom score and rescue medication use endpoints, such as symptom-free days (SFDs) and rescue medication-free days (RFD), are frequently used as clinical trial endpoints. Estimates of meaningful change for SFDs and RFDs have not been generated in pediatric populations. This research aimed to generate evidence supporting estimates of the individual within-patient changes that constitute an important or meaningful change in SFDs, RFDs, and updated estimates on the Childhood Asthma Control Test (C-ACT) in pediatric asthma populations aged 5-11 years. Methods: Semistructured, qualitative interviews were conducted with children (ages 8-11 years) who had asthma and parents/caregivers of children (4-11 years) with asthma. Before the interview (4-9 days) participants were asked to complete a morning and evening diary. Results: On average, parent/caregiver estimates of the difference in SFDs between a “very bad” and a “little bad” week for their children's asthma were largely concordant with the values reported by their children (differences of 1.8 and 1.4 SFDs, respectively). Both parents/caregivers and children were able to articulate what a meaningful level of change would be on the C-ACT at the item level. This qualitative study generated C-ACT item-level meaningful change estimates in the region of 1-3 category change, which potentially suggests that, if scaled up to represent C-ACT total score, this would lead to change estimates of 7-15 points. Conclusions: Our findings suggest that both children with asthma and parents/caregivers can quantitatively estimate and to some extent qualitatively articulate meaningful change in SFDs and RFDs.

AB - Introduction: Diary-derived symptom score and rescue medication use endpoints, such as symptom-free days (SFDs) and rescue medication-free days (RFD), are frequently used as clinical trial endpoints. Estimates of meaningful change for SFDs and RFDs have not been generated in pediatric populations. This research aimed to generate evidence supporting estimates of the individual within-patient changes that constitute an important or meaningful change in SFDs, RFDs, and updated estimates on the Childhood Asthma Control Test (C-ACT) in pediatric asthma populations aged 5-11 years. Methods: Semistructured, qualitative interviews were conducted with children (ages 8-11 years) who had asthma and parents/caregivers of children (4-11 years) with asthma. Before the interview (4-9 days) participants were asked to complete a morning and evening diary. Results: On average, parent/caregiver estimates of the difference in SFDs between a “very bad” and a “little bad” week for their children's asthma were largely concordant with the values reported by their children (differences of 1.8 and 1.4 SFDs, respectively). Both parents/caregivers and children were able to articulate what a meaningful level of change would be on the C-ACT at the item level. This qualitative study generated C-ACT item-level meaningful change estimates in the region of 1-3 category change, which potentially suggests that, if scaled up to represent C-ACT total score, this would lead to change estimates of 7-15 points. Conclusions: Our findings suggest that both children with asthma and parents/caregivers can quantitatively estimate and to some extent qualitatively articulate meaningful change in SFDs and RFDs.

UR - http://www.mendeley.com/research/pro30-economic-burden-pompe-disease-russian-federation

U2 - 10.1016/j.jval.2019.04.1663

DO - 10.1016/j.jval.2019.04.1663

M3 - тезисы

VL - 22

SP - S340

JO - Value in Health

JF - Value in Health

SN - 1098-3015

IS - Suppl.2

ER -