ArticlePediatric neurology2013
National variation in costs and mortality for leukodystrophy patients in US children's hospitals.
Article in Pediatric neurology, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
24 citing papers in PubMed, 1 synthesis or guideline pooled it, 37 citations in OpenAlex.
- Revised consensus statement on the preventive and symptomatic care of patients with leukodystrophies.Molecular genetics and metabolism · 2017Guideline
- Cost comparison by treatment arm and center-level variations in cost and inpatient days on the phase III high-risk B acute lymphoblastic leukemia trial AALL0232.Cancer medicine · 2018Trial
- The impact of vanishing white matter on unaffected family members.Orphanet journal of rare diseases · 2025Article
- Clinically Important Endpoints in Individuals With Leukodystrophy: A Multisite Study.Annals of the Child Neurology Society · 2025Article
- Healthcare utilization and disease burden in children with metachromatic leukodystrophy in Germany.Orphanet journal of rare diseases · 2025Article
- Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrations.Molecular therapy. Nucleic acids · 2025Article
- A novel variant in ARSA causes a rare phenotype of infantile metachromatic leukodystrophy in a Malian family.Neurogenetics · 2025Article
- Resource Utilization and Risk Factors for Esophageal Injury in Pediatric Esophageal Foreign Bodies.The Laryngoscope · 2024Article
- Impact on physical, social, and family functioning of patients with metachromatic leukodystrophy and their family members in Japan: A qualitative study.Molecular genetics and metabolism reports · 2024Article
- Hospitalization Burden and Incidence of Krabbe Disease.Journal of child neurology · 2022Article
- Reliability of the Telemedicine Application of the Gross Motor Function Measure-88 in Patients With Leukodystrophy.Pediatric neurology · 2021Article
- Novel and known morbidities of leukodystrophies identified using a phenome-wide association study.Neurology. Clinical practice · 2020Article
- Racial/Ethnic and Insurance Status Disparities in Distance Traveled to Access Children's Hospital Care for Severe Illness: the Case of Children with Leukodystrophies.Journal of racial and ethnic health disparities · 2020Article
- Geographic and Specialty Access Disparities in US Pediatric Leukodystrophy Diagnosis.The Journal of pediatrics · 2020Article
- Metachromatic leukodystrophy: Characterization of two (p.Leu433Val, p.Gly449Arg) arylsulfatase A mutations.Experimental and therapeutic medicine · 2019Article
- Association of Diagnosis of Leukodystrophy With Race and Ethnicity Among Pediatric and Adolescent Patients.JAMA network open · 2018Article
- Megalencephalic leukoencephalopathy with subcortical cysts: Characterization of disease variants.Neurology · 2018Observational
- The International Working Group on Neurotransmitter related Disorders (iNTD): A worldwide research project focused on primary and secondary neurotransmitter disorders.Molecular genetics and metabolism reports · 2016Article
- Article
- Emerging treatments for pediatric leukodystrophies.Pediatric clinics of North America · 2015Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors at 3 institutions in 1 country.
Funding
Abstract
backgroundInherited leukodystrophies are progressive, debilitating neurological disorders with few treatment options and high mortality rates. Our objective was to determine national variation in the costs for leukodystrophy patients and to evaluate differences in their care.
methodsWe developed an algorithm to identify inherited leukodystrophy patients in deidentified data sets using a recursive tree model based on International Classification of Disease, 9th Edition, Clinical Modification, diagnosis and procedure charge codes. Validation of the algorithm was performed independently at two institutions, and with data from the Pediatric Health Information System (PHIS) of 43 US children's hospitals, for a 7-year period between 2004 and 2010.
resultsA recursive algorithm was developed and validated, based on six International Classification of Disease, 9th Edition, Clinical Modification, codes and one procedure code that had a sensitivity up to 90% (range 61-90%) and a specificity up to 99% (range 53-99%) for identifying inherited leukodystrophy patients. Inherited leukodystrophy patients comprise 0.4% of admissions to children's hospitals and 0.7% of costs. During 7 years, these patients required $411 million of hospital care, or $131,000/patient. Hospital costs for leukodystrophy patients varied at different institutions, ranging from two to 15 times more than the average pediatric patient. There was a statistically significant correlation between higher volume and increased cost efficiency. Increased mortality rates had an inverse relationship with increased patient volume that was not statistically significant.
conclusionsWe developed and validated a code-based algorithm for identifying leukodystrophy patients in deidentified national datasets. Leukodystrophy patients account for $59 million of costs yearly at children's hospitals. Our data highlight potential to reduce unwarranted variability and improve patient care.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.