Search: onr:"swepub:oai:DiVA.org:umu-134286" >
Burden of Illness f...
Burden of Illness for Patients with Hereditary Attr Amyloidosis with Polyneuropathy Begins with Symptom Onset and Increases with Disease Progression
-
Denoncourt, R. N. (author)
-
Adams, D. (author)
-
Gonzalez-Duarte, A. (author)
-
show more...
-
O'Riordan, W. (author)
-
Yang, C. (author)
-
Yamashita, T. (author)
-
Kristen, A. (author)
-
Tournev, I (author)
-
Schmidt, H. (author)
-
Coelho, T. (author)
-
Berk, J. (author)
-
Lin, K. (author)
-
Chen, J. (author)
-
Gollob, J. (author)
-
- Suhr, Ole (author)
- Umeå universitet,Medicin
-
show less...
-
(creator_code:org_t)
- Elsevier, 2016
- 2016
- English.
-
In: Value in Health. - : Elsevier. - 1098-3015 .- 1524-4733. ; 19:7, s. A436-A436
- Related links:
-
http://www.valueinhe...
-
show more...
-
https://urn.kb.se/re...
-
https://doi.org/10.1...
-
show less...
Abstract
Subject headings
Close
- Objectives: Hereditary ATTR amyloidosis with polyneuropathy (hATTR-PN), also known as familial amyloidotic polyneuropathy (FAP), is an inherited, progressive disease leading to death within 5-15 years. hATTR-PN is due to a mutation in the transthyretin (TTR) gene. Patisiran is an investigational, RNA interference (RNAi) therapeutic targeting TTR. This abstract aims to further characterize hATTR-PN burden of illness.Methods: The ongoing, patisiran Phase 3 APOLLO trial was utilized to collect patient-reported EQ-5D, Norfolk-DN, Rasch-built Overall Disability Scale (R-ODS), and healthcare resource utilization from hATTR-PN patients with symptomatic disease.Results: APOLLO included 225 patients; median age of 62 years (range: 24-82), median neurological impairment score (NIS) of 6-141.6 and representative of the global patient population (19 countries) with a broad range of mutations and disease severities. At baseline, 57 patients had a Polyneuropathy Disability (PND) Score I and 168 patients had a PND Score ≥ II. By FAP Stage, 104 were FAP Stage 1 and 121 were FAP Stage 2. 41 patients (PND Score ≥ II) reported ≥1 hospitalization of ≥3 nights in duration due to hATTR-PN in the 12 months prior to enrollment. Mean EQ-5D scores were 0.76 (PND Score I) and 0.59 (PND Score ≥ II). Patients reported perceived health status on the EQ-VAS with mean scores of 66.9 (PND Score I) and 51.3 (PND Score ≥ II). Mean Norfolk-QoL-DN scores were 35.5 (PND Score I) and 66.0 (PND Score ≥ II). 145 patients (131 PND Score ≥ II) reported they cannot work because of hATTR-PN. Mean R-ODS scores were 40.9 and 25.9 for PND Score I and PND Score ≥ II, respectively.Conclusions: These data, from the largest controlled study of patients with hATTR-PN to date, further demonstrate that patients experience considerable burden of illness early in the course of disease and this burden increases with disease progression.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Reumatologi och inflammation (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Rheumatology and Autoimmunity (hsv//eng)
Publication and Content Type
- ref (subject category)
- art (subject category)
Find in a library
To the university's database
- By the author/editor
-
Denoncourt, R. N ...
-
Adams, D.
-
Gonzalez-Duarte, ...
-
O'Riordan, W.
-
Yang, C.
-
Yamashita, T.
-
show more...
-
Kristen, A.
-
Tournev, I
-
Schmidt, H.
-
Coelho, T.
-
Berk, J.
-
Lin, K.
-
Chen, J.
-
Gollob, J.
-
Suhr, Ole
-
show less...
- About the subject
-
- MEDICAL AND HEALTH SCIENCES
-
MEDICAL AND HEAL ...
-
and Clinical Medicin ...
-
and Rheumatology and ...
- Articles in the publication
-
Value in Health
- By the university
-
Umeå University