Patient-reported outcomes among patients with systemic mastocytosis in routine clinical practice: Results of the TouchStone SM Patient Survey.
cross-sectional studies
disease burden
myeloid neoplasm
myeloproliferative neoplasm
patient-reported outcomes
systemic mastocytosis
Journal
Cancer
ISSN: 1097-0142
Titre abrégé: Cancer
Pays: United States
ID NLM: 0374236
Informations de publication
Date de publication:
10 2022
10 2022
Historique:
revised:
22
04
2022
received:
10
02
2022
accepted:
13
05
2022
pubmed:
24
8
2022
medline:
28
9
2022
entrez:
23
8
2022
Statut:
ppublish
Résumé
Systemic mastocytosis (SM) is a rare clonal neoplasm driven by KIT D816V and other mutations. Data were collected from the patient perspective on disease burden and included an SM-specific symptom assessment tool. US adults aged 18 years and older with a self-reported SM diagnosis completed an online TouchStone SM Patient Survey of 100 items, including the 12-item Short-Form Health Survey, the Indolent Systemic Mastocytosis Symptom Assessment Form, and the Work Productivity and Activity Impairment Questionnaire, as well as questions about SM diagnosis, the impact of SM on daily activities, work impairment, and health care use. The results were analyzed using descriptive statistics. Fifty-six individuals completed the survey (89% women; median age, 48 years; mean time since diagnosis, 6.7 years), reporting indolent SM (66%), aggressive SM (9%), smoldering SM (5%), and unknown SM subtype (18%). Over a 1-year recall, respondents reported seeking emergency care for anaphylaxis (30%) and taking three or more prescription medications (52%) for SM. Over one half of patients (54%) reduced their work hours because of SM, and 64% avoided leaving home because of symptoms. A majority of respondents (93%) had experienced ≥10 SM-related symptoms, noting that the most bothersome were anaphylactic episodes (18%), abdominal/stomach pain (16%), diarrhea/loose stools (13%), and fatigue (11%). Whereas an Indolent Systemic Mastocytosis Symptom Assessment Form-derived total symptom score of 28 is used to indicate moderate-to-severe symptoms, the mean total symptom score was 52.7. Mental and physical component summary scores from the 12-item Short-Form Health Survey were below population norms. Patients who were surveyed reported substantial symptom burden and unmet needs because of SM, as evidenced by seeking emergency care and reporting bothersome symptoms, poor quality of life, and reduced work hours and productivity. The objective of this research was to understand the burden and unmet needs in the rare disease of systemic mastocytosis (SM) to guide future care. Fifty-six patients completed an online survey containing questions about their diagnosis, medications, health care use, quality of life, and SM symptoms. The results demonstrated that SM is associated with severe and burdensome symptoms, anaphylactic events, emergency department visits, use of multiple medications, reduced ability to work, and poor physical and psychological quality of life. These findings suggest the need for future advances to address unmet needs in patients affected by SM.
Sections du résumé
BACKGROUND
Systemic mastocytosis (SM) is a rare clonal neoplasm driven by KIT D816V and other mutations. Data were collected from the patient perspective on disease burden and included an SM-specific symptom assessment tool.
METHODS
US adults aged 18 years and older with a self-reported SM diagnosis completed an online TouchStone SM Patient Survey of 100 items, including the 12-item Short-Form Health Survey, the Indolent Systemic Mastocytosis Symptom Assessment Form, and the Work Productivity and Activity Impairment Questionnaire, as well as questions about SM diagnosis, the impact of SM on daily activities, work impairment, and health care use. The results were analyzed using descriptive statistics.
RESULTS
Fifty-six individuals completed the survey (89% women; median age, 48 years; mean time since diagnosis, 6.7 years), reporting indolent SM (66%), aggressive SM (9%), smoldering SM (5%), and unknown SM subtype (18%). Over a 1-year recall, respondents reported seeking emergency care for anaphylaxis (30%) and taking three or more prescription medications (52%) for SM. Over one half of patients (54%) reduced their work hours because of SM, and 64% avoided leaving home because of symptoms. A majority of respondents (93%) had experienced ≥10 SM-related symptoms, noting that the most bothersome were anaphylactic episodes (18%), abdominal/stomach pain (16%), diarrhea/loose stools (13%), and fatigue (11%). Whereas an Indolent Systemic Mastocytosis Symptom Assessment Form-derived total symptom score of 28 is used to indicate moderate-to-severe symptoms, the mean total symptom score was 52.7. Mental and physical component summary scores from the 12-item Short-Form Health Survey were below population norms.
CONCLUSIONS
Patients who were surveyed reported substantial symptom burden and unmet needs because of SM, as evidenced by seeking emergency care and reporting bothersome symptoms, poor quality of life, and reduced work hours and productivity.
LAY SUMMARY
The objective of this research was to understand the burden and unmet needs in the rare disease of systemic mastocytosis (SM) to guide future care. Fifty-six patients completed an online survey containing questions about their diagnosis, medications, health care use, quality of life, and SM symptoms. The results demonstrated that SM is associated with severe and burdensome symptoms, anaphylactic events, emergency department visits, use of multiple medications, reduced ability to work, and poor physical and psychological quality of life. These findings suggest the need for future advances to address unmet needs in patients affected by SM.
Identifiants
pubmed: 35996873
doi: 10.1002/cncr.34420
pmc: PMC9804682
doi:
Substances chimiques
Proto-Oncogene Proteins c-kit
EC 2.7.10.1
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
3691-3699Commentaires et corrections
Type : CommentIn
Informations de copyright
© 2022 The Authors. Cancer published by Wiley Periodicals LLC on behalf of American Cancer Society.
Références
J Allergy Clin Immunol Pract. 2019 Jan;7(1):81-87
pubmed: 30416055
J Pharm Pract. 2021 Jun;34(3):365-371
pubmed: 31451091
Am J Hematol. 2021 Apr 1;96(4):508-525
pubmed: 33524167
Fam Pract. 2021 Sep 25;38(5):699-702
pubmed: 34213547
Cancer. 2015 Aug 15;121(16):2831-9
pubmed: 25891437
Front Pharmacol. 2020 Apr 14;11:443
pubmed: 32346366
J Allergy Clin Immunol. 2022 Feb;149(2):728-735.e2
pubmed: 34174297
Blood. 2009 Jun 4;113(23):5727-36
pubmed: 19363219
Orphanet J Rare Dis. 2021 Oct 18;16(1):434
pubmed: 34663404
Lancet Haematol. 2019 Dec;6(12):e638-e649
pubmed: 31676322
Med Care. 1996 Mar;34(3):220-33
pubmed: 8628042
J Allergy Clin Immunol Pract. 2019 Apr;7(4):1097-1106
pubmed: 30961835
Allergy. 2016 Nov;71(11):1585-1593
pubmed: 27089859
Front Med (Lausanne). 2017 Jul 20;4:110
pubmed: 28775983
Br J Haematol. 2014 Aug;166(4):521-8
pubmed: 24761987
BMC Cancer. 2016 Feb 27;16:167
pubmed: 26922064
N Engl J Med. 2015 Jul 9;373(2):163-72
pubmed: 26154789
Leuk Lymphoma. 2021 Sep;62(9):2253-2260
pubmed: 33749512
J Allergy Clin Immunol Pract. 2014 Jan-Feb;2(1):70-6
pubmed: 24565772
Cancer. 2017 Feb 1;123(3):449-458
pubmed: 27690182
J Allergy Clin Immunol. 2020 Aug;146(2):356-366.e4
pubmed: 32437738
J Invest Dermatol. 2012 Apr;132(4):1111-6
pubmed: 22205305
J Patient Exp. 2021 Nov 23;8:23743735211059053
pubmed: 34888413
Blood. 2016 May 19;127(20):2391-405
pubmed: 27069254
Expert Rev Hematol. 2021 Aug;14(8):687-696
pubmed: 34289787
J Am Med Inform Assoc. 2019 Aug 1;26(8-9):703-713
pubmed: 31081898
Pharmacoeconomics. 1993 Nov;4(5):353-65
pubmed: 10146874
Orphanet J Rare Dis. 2021 Oct 9;16(1):414
pubmed: 34627355
Immunol Allergy Clin North Am. 2018 Aug;38(3):505-525
pubmed: 30007467
PLoS One. 2020 Dec 31;15(12):e0243562
pubmed: 33382745