Healthcare Resource Use Among Children With Mucopolysaccharidosis Type III MPS III: A Retrospective Claims Database Analysis in the US
Author(s)
Manpreet K. Sidhu, BA, MBA, PhD1, Loreina Guo, MS2, Lizheng Shi, PhD3, Daniel Garcia, MD1.
1Ultragenyx Pharmaceutical Inc., Novato, CA, USA, 2NouStarX, Short Hills, NJ, USA, 3Tulane University School of Public Health and Tropical Medicine, New Orleans, LA, USA.
1Ultragenyx Pharmaceutical Inc., Novato, CA, USA, 2NouStarX, Short Hills, NJ, USA, 3Tulane University School of Public Health and Tropical Medicine, New Orleans, LA, USA.
OBJECTIVES: MPS III, also known as Sanfilippo syndrome, is a rare, inherited lysosomal storage disorder with a global incidence of 1:100,000 that is characterized by developmental regression in early childhood; behavioral challenges, including hyperactivity and sleep disturbance; and early death. This retrospective cohort study evaluated healthcare resource use (HCRU) in US children with MPS III.
METHODS: The IQVIA PharMetrics® Plus database was searched for pediatric patients diagnosed with MPS III, defined as ≥1 ICD-10-CM Diagnosis Code E76.22 in any billing position of a claim from 01Jan2016 to 31Mar2024. Patients had ≥12 months of continuous enrollment (CE) during the study period. Patients were excluded if there was any evidence of clinical trial participation, pregnancy, or data quality issues. The control cohort, patients without the ICD-10 code for MPS III, were matched on age, gender, payer type on CE start month, and CE start year (1:20 of MPS III to the control group). Supportive or adjunctive therapies, clinical manifestations, and HCRU were assessed during all time periods available after the first diagnosis code date and were identified using ICD-10-CM Diagnosis Codes in any care setting.
RESULTS: Of 113 children with MPS III, 35 (30.9%) were ≤5 years, 27 (24.0%) were between 6-9 years, and 51 (45.1%) were 10-17 years; 56 (49.6%) were female. Compared to the control cohort, patients with MPS III had increased use of anti-epileptic medications (50% vs 2%), pain medications (36% vs 15%), and sleep medications (11% vs 0%). Mean (SD) annualized average number of hospitalizations was 0.26 (0.62) in MPS III with length of stay (LOS) of 7.2 (21.17) days versus 0.02 (0.17) in the control group with 6.38 (10.85) days LOS.
CONCLUSIONS: This study demonstrated that patients with MPS III experienced higher use of supportive or adjunctive therapies and greater health care resource use than comparator patients.
METHODS: The IQVIA PharMetrics® Plus database was searched for pediatric patients diagnosed with MPS III, defined as ≥1 ICD-10-CM Diagnosis Code E76.22 in any billing position of a claim from 01Jan2016 to 31Mar2024. Patients had ≥12 months of continuous enrollment (CE) during the study period. Patients were excluded if there was any evidence of clinical trial participation, pregnancy, or data quality issues. The control cohort, patients without the ICD-10 code for MPS III, were matched on age, gender, payer type on CE start month, and CE start year (1:20 of MPS III to the control group). Supportive or adjunctive therapies, clinical manifestations, and HCRU were assessed during all time periods available after the first diagnosis code date and were identified using ICD-10-CM Diagnosis Codes in any care setting.
RESULTS: Of 113 children with MPS III, 35 (30.9%) were ≤5 years, 27 (24.0%) were between 6-9 years, and 51 (45.1%) were 10-17 years; 56 (49.6%) were female. Compared to the control cohort, patients with MPS III had increased use of anti-epileptic medications (50% vs 2%), pain medications (36% vs 15%), and sleep medications (11% vs 0%). Mean (SD) annualized average number of hospitalizations was 0.26 (0.62) in MPS III with length of stay (LOS) of 7.2 (21.17) days versus 0.02 (0.17) in the control group with 6.38 (10.85) days LOS.
CONCLUSIONS: This study demonstrated that patients with MPS III experienced higher use of supportive or adjunctive therapies and greater health care resource use than comparator patients.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
RWD91
Topic
Economic Evaluation, Real World Data & Information Systems
Topic Subcategory
Health & Insurance Records Systems
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases