Content validity of patient-reported outcomes for use in lower-risk myelodysplastic syndromes.
FACT-An
Health-related quality of life
Myelodysplastic syndrome
Patient reported outcomes
QUALMS
Journal
Journal of patient-reported outcomes
ISSN: 2509-8020
Titre abrégé: J Patient Rep Outcomes
Pays: Germany
ID NLM: 101722688
Informations de publication
Date de publication:
26 Aug 2020
26 Aug 2020
Historique:
received:
11
08
2019
accepted:
13
08
2020
entrez:
28
8
2020
pubmed:
28
8
2020
medline:
28
8
2020
Statut:
epublish
Résumé
The lower-risk (low and intermediate-1 risk based on IPSS) myelodysplastic syndrome (MDS) has a negative impact on patients' health-related quality of life (HRQoL). Patient Reported Outcomes (PROs) instruments, which are used to collect patients' HRQoL data, should have established content validity in the target population to ensure that the instrument is comprehensive and comprehensible. The present study was conducted to evaluate the content validity of the Quality of Life in Myelodysplasia Scale (QUALMS) and the Functional Assessment of Cancer Therapy-Anemia (FACT-An) PRO instruments in patients with lower-risk MDS. In this cross-sectional, qualitative study, 16 patients aged ≥18 years with lower-risk MDS, who were RBC transfusion dependent, literate and fluent in US-English were interviewed. Interviews were semi-structured comprising of two parts: concept elicitation (CE) explored symptoms and impacts important to patients, and cognitive debriefing (CD) assessed understanding and relevance of the QUALMS and FACT-An. A conceptual model was developed, which was used to map the concepts that emerged during CE onto the QUALMS and FACT-An to assess concept coverage and suitability of the instruments. The median age of participants was 67.5 years (range: 51-91), with half being female (n = 8). Nine (56.2%) participants had intermediate-1-risk MDS and 10 (62.5%) were relapsed or refractory to erythropoiesis-stimulating agent treatment. Fatigue/tiredness (100.0%), shortness of breath (87.5%), weakness (81.2%), and low energy (75.0%) were reported most commonly and were the most bothersome symptoms as well. Of seven high-level HRQoL domains identified, activities of daily living (n = 16, 100.0%), physical functioning (n = 15, 93.8%), emotional wellbeing (n = 13, 81.3%), social functioning (n = 12, 75.0%), sleep disturbance (n = 9, 56.3%), and impact on work (n = 9, 56.3%) were the most commonly reported. For CD, the QUALMS and FACT-An were found to be mostly relevant and very well understood; response options were easy to use, and recall period was appropriate. Both QUALMS and FACT-An demonstrated a strong face and content validity in patients with lower-risk MDS, suggesting that these instruments are appropriate for assessing HRQoL in this population.
Sections du résumé
BACKGROUND
BACKGROUND
The lower-risk (low and intermediate-1 risk based on IPSS) myelodysplastic syndrome (MDS) has a negative impact on patients' health-related quality of life (HRQoL). Patient Reported Outcomes (PROs) instruments, which are used to collect patients' HRQoL data, should have established content validity in the target population to ensure that the instrument is comprehensive and comprehensible. The present study was conducted to evaluate the content validity of the Quality of Life in Myelodysplasia Scale (QUALMS) and the Functional Assessment of Cancer Therapy-Anemia (FACT-An) PRO instruments in patients with lower-risk MDS.
METHODS
METHODS
In this cross-sectional, qualitative study, 16 patients aged ≥18 years with lower-risk MDS, who were RBC transfusion dependent, literate and fluent in US-English were interviewed. Interviews were semi-structured comprising of two parts: concept elicitation (CE) explored symptoms and impacts important to patients, and cognitive debriefing (CD) assessed understanding and relevance of the QUALMS and FACT-An. A conceptual model was developed, which was used to map the concepts that emerged during CE onto the QUALMS and FACT-An to assess concept coverage and suitability of the instruments.
RESULTS
RESULTS
The median age of participants was 67.5 years (range: 51-91), with half being female (n = 8). Nine (56.2%) participants had intermediate-1-risk MDS and 10 (62.5%) were relapsed or refractory to erythropoiesis-stimulating agent treatment. Fatigue/tiredness (100.0%), shortness of breath (87.5%), weakness (81.2%), and low energy (75.0%) were reported most commonly and were the most bothersome symptoms as well. Of seven high-level HRQoL domains identified, activities of daily living (n = 16, 100.0%), physical functioning (n = 15, 93.8%), emotional wellbeing (n = 13, 81.3%), social functioning (n = 12, 75.0%), sleep disturbance (n = 9, 56.3%), and impact on work (n = 9, 56.3%) were the most commonly reported. For CD, the QUALMS and FACT-An were found to be mostly relevant and very well understood; response options were easy to use, and recall period was appropriate.
CONCLUSION
CONCLUSIONS
Both QUALMS and FACT-An demonstrated a strong face and content validity in patients with lower-risk MDS, suggesting that these instruments are appropriate for assessing HRQoL in this population.
Identifiants
pubmed: 32851569
doi: 10.1186/s41687-020-00235-4
pii: 10.1186/s41687-020-00235-4
pmc: PMC7450032
doi:
Types de publication
Journal Article
Langues
eng
Pagination
69Références
Cancer Genet Cytogenet. 1985 Jul;17(3):189-255
pubmed: 3891074
Eur J Oncol Nurs. 2015 Apr;19(2):99-106
pubmed: 25488465
Qual Life Res. 2010 Oct;19(8):1087-96
pubmed: 20512662
J Natl Cancer Inst. 2008 Nov 5;100(21):1542-51
pubmed: 18957672
Haematologica. 2013 Feb;98(2):208-16
pubmed: 22875615
Oncology (Williston Park). 2002 Sep;16(9 Suppl 10):125-32
pubmed: 12380962
J Geriatr Oncol. 2015 Jul;6(4):288-98
pubmed: 26073533
Cancer Med. 2019 Feb;8(2):543-553
pubmed: 30632713
Curr Med Res Opin. 2009 Apr;25(4):929-42
pubmed: 19257798
Blood. 2013 Oct 24;122(17):2943-64
pubmed: 23980065
Br J Haematol. 2015 Aug;170(3):372-83
pubmed: 25907546
J Patient Rep Outcomes. 2018 Sep 06;2:41
pubmed: 30238086
Value Health. 2018 Jul;21(7):839-842
pubmed: 30005756
Leuk Res. 2013 Mar;37(3):259-65
pubmed: 23273538
Crit Rev Oncol Hematol. 2015 Dec;96(3):542-54
pubmed: 26324461
Leuk Res. 2008 May;32(5):691-8
pubmed: 18054795
Leukemia. 2008 Mar;22(3):538-43
pubmed: 18079733
Leukemia. 2019 Mar;33(3):570-575
pubmed: 30700839
J Transl Int Med. 2017 Sep 30;5(3):139-143
pubmed: 29085786
Ann Med. 2014 Aug;46(5):283-9
pubmed: 24716735
Pharmacoeconomics. 2008;26(5):363-70
pubmed: 18429654
J Clin Oncol. 2013 Jul 10;31(20):2548-53
pubmed: 23733767
Cancer. 2018 Mar 15;124(6):1251-1259
pubmed: 29231969
Haematologica. 2016 Jun;101(6):781-8
pubmed: 26944474
JAMA Oncol. 2015 Nov;1(8):1051-9
pubmed: 26270597
J Clin Oncol. 2007 Aug 10;25(23):3503-10
pubmed: 17687155
Leuk Res. 2008 Jul;32(7):1049-53
pubmed: 18191202
Qual Life Res. 2012 Aug;21(6):1013-20
pubmed: 21909804
Ecancermedicalscience. 2019 Mar 28;13:913
pubmed: 31123496
Clin Cancer Res. 2006 Jan 1;12(1):5-10
pubmed: 16397017
Br J Haematol. 2003 Apr;121(2):270-4
pubmed: 12694248
Am J Hematol. 2016 Jan;91(1):76-89
pubmed: 26769228
Am J Blood Res. 2012;2(2):136-47
pubmed: 22762033
Br J Haematol. 2016 Jul;174(1):88-101
pubmed: 26991631
Br J Haematol. 2003 Mar;120(6):1037-46
pubmed: 12648074
Curr Hematol Malig Rep. 2015 Sep;10(3):272-81
pubmed: 26134527
Ann Hematol. 2014 Sep;93(9):1523-9
pubmed: 24711171
Lancet Oncol. 2015 Nov;16(15):1506-1514
pubmed: 26404501
Hemasphere. 2019 Oct 30;3(6):e314
pubmed: 31976486
Leuk Res. 2005 Oct;29(10):1217-9
pubmed: 16111537
Health Qual Life Outcomes. 2006 Oct 11;4:79
pubmed: 17034633
Value Health. 2007 Nov-Dec;10 Suppl 2:S125-37
pubmed: 17995471
Blood. 2014 Jan 16;123(3):451-2
pubmed: 24434998
Value Health. 2009 Nov-Dec;12(8):1075-83
pubmed: 19804437
Leukemia. 2018 Jun;32(6):1380-1392
pubmed: 29572506
Blood. 2000 Dec 1;96(12):3671-4
pubmed: 11090046
J Pain Symptom Manage. 2015 Mar;49(3):513-20
pubmed: 25128608