Durable Medical Equipment and Ancillary Service Use in Rett Syndrome Management: A Global Comprehensive Review of Literature
Author(s)
Nazia Rashid, MS, PharmD1, Safiuddin Syed, PhD2, Kitty Rajagopalan, BS, MS, PhD3, Mirko Sikirica, PharmD1, Ismaeel U. Yunusa, PharmD, PhD4.
1Medical Affaits, ACADIA Pharmaceuticals, San Diego, CA, USA, 2Anlitiks, Windermere, FL, USA, 3Anlitiks, WINDERMERE, FL, USA, 4University of South Carolina Student Chapter, Columbia, SC, USA.
1Medical Affaits, ACADIA Pharmaceuticals, San Diego, CA, USA, 2Anlitiks, Windermere, FL, USA, 3Anlitiks, WINDERMERE, FL, USA, 4University of South Carolina Student Chapter, Columbia, SC, USA.
OBJECTIVES: To conduct a global comprehensive literature review (CLR) to better understand the prevalence rates of additional services used among individuals with RETT (RTT) syndrome.
METHODS: A CLR was conducted using searches of Medline, Embase, Cochrane Library, Trial Registries, and other evidence platforms from January 1, 2000, to July 31, 2024. English language articles of RTT from any country that reported the use of DME (e.g., wheelchair, gastrostomy, etc.) and ancillary therapy services [physical (PT), occupational (OTD), speech therapy (ST)] were included and reviewed. Rates (%) were reported as prevalence rates.
RESULTS: Among RTT patients, 26 studies were included in this CLR: 12 studies reported gastrostomy use (rates 3.7%-38.0%) and 3 studies reported enteral feeding (rates 19.5%-52.0%). DME, specifically wheelchairs, were utilized by 33.3%-83.5% of RTT patients in 2 studies. Rates of ancillary therapy service use was reported among various 2 studies as follows: PT use (24.4%-92.2%), OT use (11.5%-40.9%), and ST use (13.3%-17.0%). Feeding assistance was examined in 2 studies (37.9%-92.6%). Additionally, we found scoliosis surgeries were performed in 1.2%-55.5% of RTT patients across 5 studies. Music therapy and hospice care were utilized by 38% and 24.9% of RTT patients, respectively.
CONCLUSIONS: Management of RTT necessitates ongoing monitoring and requires various therapeutic and supportive interventions to optimize health and well-being. This global CLR demonstrates that a high proportion of patients require wheelchairs, enteral feeding, and physical therapy as a part of RTT management.
METHODS: A CLR was conducted using searches of Medline, Embase, Cochrane Library, Trial Registries, and other evidence platforms from January 1, 2000, to July 31, 2024. English language articles of RTT from any country that reported the use of DME (e.g., wheelchair, gastrostomy, etc.) and ancillary therapy services [physical (PT), occupational (OTD), speech therapy (ST)] were included and reviewed. Rates (%) were reported as prevalence rates.
RESULTS: Among RTT patients, 26 studies were included in this CLR: 12 studies reported gastrostomy use (rates 3.7%-38.0%) and 3 studies reported enteral feeding (rates 19.5%-52.0%). DME, specifically wheelchairs, were utilized by 33.3%-83.5% of RTT patients in 2 studies. Rates of ancillary therapy service use was reported among various 2 studies as follows: PT use (24.4%-92.2%), OT use (11.5%-40.9%), and ST use (13.3%-17.0%). Feeding assistance was examined in 2 studies (37.9%-92.6%). Additionally, we found scoliosis surgeries were performed in 1.2%-55.5% of RTT patients across 5 studies. Music therapy and hospice care were utilized by 38% and 24.9% of RTT patients, respectively.
CONCLUSIONS: Management of RTT necessitates ongoing monitoring and requires various therapeutic and supportive interventions to optimize health and well-being. This global CLR demonstrates that a high proportion of patients require wheelchairs, enteral feeding, and physical therapy as a part of RTT management.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EPH66
Topic
Clinical Outcomes, Epidemiology & Public Health, Health Service Delivery & Process of Care
Disease
Rare & Orphan Diseases