Intracerebroventricular Cerliponase Alfa for Neuronal Ceroid Lipofuscinosis Type 2 Disease: Clinical Practice Considerations From US Clinics.

BACKGROUND: Neuronal ceroid lipofuscinosis type 2 or CLN2 disease is a rare, autosomal recessive, neurodegenerative lysosomal storage disorder caused by tripeptidyl peptidase 1 deficiency. Cerliponase alfa, a recombinant human tripeptidyl peptidase 1 enzyme, is the first and only approved treatment for CLN2 disease and the first approved enzyme replacement therapy administered via intracerebroventricular infusion. METHODS: A meeting of health care professionals from US institutions with experience in cerliponase alfa treatment of children with CLN2 disease was held in November 2018. Key common practices were identified, and later refined during the drafting of this article, that facilitate safe chronic administration of cerliponase alfa. RESULTS: Key practices include developing a multidisciplinary team of clinicians, pharmacists, and coordinators, and institution-specific processes. Infection risk may be reduced through strict aseptic techniques and minimizing connections and disconnections during infusion. The impact of intracerebroventricular device design on port needle stability during extended intracerebroventricular infusion is a critical consideration in device selection. Monitoring for central nervous system infection is performed at each patient contact, but with flexibility in the degree of monitoring. Although few institutions had experienced positive cerebrospinal fluid test results, the response to a positive cerebrospinal fluid culture should be determined on a case-by-case basis, and the intracerebroventricular device should be removed if cerebrospinal fluid infection is confirmed. CONCLUSIONS: The key common practices and flexible practices used by institutions with cerliponase alfa experience may assist other institutions in process development. Continued sharing of experiences will be essential for developing standards and patient care guidelines.

Pediatric retropharyngeal abscesses: a national perspective.

OBJECTIVES: To determine the resource utilization and national variation in the management of pediatric retropharyngeal abscesses. METHODS: The Kids' Inpatient Database (KID) 2003 was analyzed. International Classification of Diseases, Ninth Revision code 478.24 was the inclusion criteria. RESULTS: One thousand three hundred and twenty-one admissions with retropharyngeal abscess were sampled from the KID in 2003; there were no deaths. The mean age of patients was 5.1 years (S.D. 4.4 years); 63% were male. Of all admissions, 563 (43%) patients underwent surgical drainage of their infection; surgical patients had longer length of stays and total charges than patients managed medically. The average state spending per admission varied from $5126 (Utah) to $27,776 (California). There was seasonal variation in admissions with the highest percentage of admissions occurring in March (10.7%) and lowest in August (3.8%). Indicators of increased resource utilization included age (older patients), increased length of stay, non-elective admission, discharge quarter, and number of other diagnoses on record. There is a statistically significant decrease in the length of stay and total charges in patients admitted in the Midwest compared to other regions of the country. CONCLUSIONS: This study demonstrates national demographics and normative data on a commonly treated pediatric disease process, retropharyngeal space infections. The average demographic of such a patient is a 5-year-old male from an urban location admitted in a non-elective fashion via the emergency department. The mean total charges were $16,377; 90% of admissions had total charges less than $28,511. Patients who underwent surgical procedures had mean total charges of $22,013. There exists significant national variation in resource utilization for this commonly treated disease process.

Hospital charges of potentially preventable pediatric hospitalizations.

OBJECTIVES: Reducing the number of preventable hospitalizations represents a possible source of health care savings. However, the current literature lacks a description of the extent of potentially preventable pediatric hospitalizations. The study objectives are to (1) identify the charges and (2) demographic characteristics associated with potentially preventable pediatric hospitalizations. METHODS: Secondary analysis of the 2006 Kids' Inpatient Database (weighted N = 7,558,812). International Classification of Diseases, Ninth Revision, Clinical Modification codes for 16 previously validated pediatric ambulatory care-sensitive (ACS) conditions identified potentially preventable hospitalizations; seven additional conditions reflected updated care guidelines. Outcome variables included number of admissions, hospitalization days, and hospital charges. Demographic and diagnostic variables associated with an ACS condition were compared with regression analyses by the use of appropriate person-level weights. RESULTS: Pediatric ACS hospitalizations totaled $4.05B in charges and 1,087,570 hospitalization days in 2006. Two respiratory conditions-asthma and bacterial pneumonia-comprised 48.4% of ACS hospital charges and 46.7% of ACS hospitalization days. In multivariate analysis, variables associated with an ACS condition included: male gender (odds ratio [OR] 1.10; 95% confidence interval [95% CI] 1.07-1.13); race/ethnicity of black (OR 1.22; 95% CI 1.16-1.27) or Hispanic (OR 1.12; 95% CI 1.06-1.18); and emergency department as admission source (OR 1.37; 95% CI 1.27-1.48). CONCLUSIONS: Respiratory conditions comprised the largest proportion of potentially preventable pediatric hospitalizations, totaling as much as $1.96B in hospital charges. Children hospitalized with an ACS condition tend to be male, non-white, and admitted through the emergency department. Future research to prevent pediatric hospitalizations should examine targeted interventions in the primary care setting, specifically around respiratory conditions and minority populations.