R Package for Pediatric Complex Chronic Condition Classification
Identification of children with complex chronic conditions (CCCs) is necessary to improve health care delivery and perform clinical research, because this patient population uses significant inpatient and outpatient medical resources.1 The original CCC classification was published in 2000.2 A second version was published in 2014 to reflect additions to the International Classification of Diseases system and the US adoption of the International Statistical Classification of Diseases and Related Health Problems, Tenth Revision.3 The CCC classification is widely used in research (currently cited in more than 100 peer-reviewed journal publications). However, the current approach to assigning the CCC categories in health care–related data sets is limited by proprietary software and computational inefficiency. SAS and Stata software to assign CCC categories were published as appendices to the 2014 update,3 but not all investigators have access to these statistical packages. In addition, increasingly large data sets are available to investigators. Although the data processing capability of individual computers continues to improve, the SAS and Stata software can take significant time to run on data sets with millions of observations. The objective of this project was to develop computationally efficient software to generate the CCC categories using R, a free, open-source statistical environment.4 We then compared the SAS, Stata, and R software with respect to accuracy and speed of classification on a typical desktop system.
Gordon LB; Shappell H; Massaro J; D'Agostino Sr RB; Brazier J; Campbell SE; Kleinman ME; Kieran MW
JAMA.
2018
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1001/jamapediatrics.2018.0256" target="_blank" rel="noreferrer noopener">10.1001/jamapediatrics.2018.0256</a>
Recent trends in cerebral palsy survival. Part II: individual survival prognosis
adolescent; Child; Female; Humans; infant; Male; Young Adult; Adult; mortality; Prognosis; Disabled Persons; California; Cerebral Palsy; Kaplan-Meier Estimate; Preschool; Life Expectancy
AIM: The aim of the study was to determine survival probabilities and life expectancies for individuals with cerebral palsy based on data collected over a 28-year period in California. METHOD: We identified all individuals with cerebral palsy, aged 4 years or older, who were clients of the California Department of Developmental Services between 1983 and 2010. Kaplan-Meier survival curves were constructed for 4-year-old children, and the estimated survival probabilities were adjusted to reflect trends in mortality by calendar year. For persons aged 15, 30, 45, and 60 years, separate Poisson regression models were used to estimate age-, sex-, and disability-specific mortality rates. These mortality rates were adjusted to reflect trends of improved survival, and life expectancies were obtained using life table methods. RESULTS: The sample comprised 16,440, 14,609, 11,735, 7023, and 2375 persons at ages 4, 15, 30, 45, and 60 years, respectively. In 1983, 50% of 4-year-old children who did not lift their heads in the prone position and were tube fed lived to age 10.9 years. By 2010, the median age at death had increased to 17.1 years. In ambulatory children the probability of survival to adulthood did not change by more than 1%. Life expectancies for adolescents and adults were lower for those with more severe limitations in motor function and feeding skills, and decreased with advancing age. Life expectancies for tube-fed adolescents and adults increased by 1 to 3 years, depending on age and pattern of disability, over the course of the study period. INTERPRETATION: Over the past three decades in California there have been significant improvements in the survival of children with very severe disabilities. There have also been improvements to the life expectancy of tube-fed adults, though to a lesser extent than in children.
2014-11
Brooks JC; Strauss DJ; Shavelle RM; Tran Linh M; Rosenbloom L; Wu YW
Developmental Medicine And Child Neurology
2014
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
Journal Article
<a href="http://doi.org/10.1111/dmcn.12519" target="_blank" rel="noreferrer">10.1111/dmcn.12519</a>
The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UK
Child; Humans; Adult; Prevalence; Kaplan-Meier Estimate; Phenotype; Demography; Registries; Medical; adolescent; Societies; England/epidemiology; Mucopolysaccharidosis I/epidemiology/mortality/physiopathology; Wales/epidemiology
BACKGROUND: Mucopolysaccharidosis type I (MPS I) is a rare lysosomal storage disease subdivided into three phenotypes of increasing severity: Scheie, Hurler-Scheie and Hurler. To gauge the effectiveness of treatments and to determine the load likely to fall on health-care systems, it is necessary to understand the prevalence and natural progression of the disease especially with regard to life-expectancy. In general such data on the natural history of lysosomal storage diseases is sparse. METHODS: Analysis of prevalence and patient survival in MPS I disease using a unique longitudinal data set initiated and maintained over a period of more than 20 years by the Society for Mucopolysaccharide Diseases (UK). RESULTS: The birth prevalence of MPS I in England and Wales over the period 1981 to 2003 was 1.07/100,000 births and within +/- 5% of estimates reported in several studies that examined reasonably large populations. The median survival for MPS I patients (including all phenotypes irrespective of various treatments) was found by Kaplan-Meier analysis to be 11.6 years. This result was driven by the relatively poor survival of patients with the Hurler phenotype who, irrespective of any treatments received, had a median survival of 8.7 years; when censoring for receipt of bone marrow transplant (BMT) was implemented median survival of Hurler patients was diminished to 6.8 years. The difference between these survival curves was statistically significant by log rank test and can be attributed to beneficial effects of BMT and or selection of patients with superior prognosis for intervention with BMT. Survival curves for Hurler patients who received and did not receive BMT were very different. Probability of survival at 2 year after BMT was ~68% and was similar to this after 5 years (66%) and ten years (64%); the mean age of Hurler patients at receipt of BMT was 1.33 years (range 0.1 to 3 years). Follow up was insufficient to determine median survival of the milder phenotypes however, unsurprisingly, this was clearly superior to that for Hurler patients. CONCLUSION: The birth prevalence of MPS I in England and Wales is 1.07/100,000 and the median survival for MPS I patients is 11.6 years.
2008
Moore D; Connock MJ; Wraith E; Lavery C
Orphanet Journal Of Rare Diseases
2008
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
Journal Article
<a href="http://doi.org/10.1186/1750-1172-3-24" target="_blank" rel="noreferrer">10.1186/1750-1172-3-24</a>
Prognostic factors for survival in 676 consecutive patients with newly diagnosed primary glioblastoma
adolescent; Female; Humans; Male; Adult; Prognosis; Aged; Middle Aged; Treatment Outcome; Kaplan-Meier Estimate; Antineoplastic Agents; Combined Modality Therapy; 80 and over; Brain neoplasms; Disease-Free Survival; Local; Glioblastoma; Neoplasm Recurrence; Neurosurgical Procedures; Radiotherapy
Reliable data on large cohorts of patients with glioblastoma are needed because such studies differ importantly from trials that have a strong bias toward the recruitment of younger patients with a higher performance status. We analyzed the outcome of 676 patients with histologically confirmed newly diagnosed glioblastoma who were treated consecutively at a single institution over a 7-year period (1997-2003) with follow-up to April 30, 2006. Survival probabilities were 57% at 1 year, 16% at 2 years, and 7% at 3 years. Progression-free survival was 15% at 1 year. Prolongation of survival was significantly associated with surgery in patients with a good performance status, whatever the patient's age, with an adjusted hazard ratio of 0.55 (p < 0.001) or a 45% relative decrease in the risk of death. Radiotherapy and chemotherapy improved survival, with adjusted hazard ratios of 0.61 (p = 0.001) and 0.89 (p = 0.04), respectively, regardless of age, performance status, or residual tumor volume. Recurrence occurred in 99% of patients throughout the follow-up. Reoperation was performed in one-fourth of these patients but was not effective, whether performed within 9 months (hazard ratio, 0.86; p = 0.256) or after 9 months (hazard ratio, 0.98; p = 0.860) of initial surgery, whereas second-line chemotherapy with procarbazine, lomustine, and vincristine (PCV) or with temozolomide improved survival (hazard ratio, 0.77; p = 0.008). Surgery followed by radiotherapy and chemotherapy should be considered in all patients with glioblastoma, and these treatments should not be withheld because of increasing age alone. The benefit of second surgery at recurrence is uncertain, and new trials are needed to assess its effectiveness. Chemotherapy with PCV or temozolomide seems to be a reasonable option at tumor recurrence.
2008-02
Filippini G; Falcone C; Boiardi A; Broggi G; Bruzzone MG; Caldiroli D; Farina R; Farinotti M; Fariselli L; Finocchiaro G; Giombini S; Pollo B; Savoiardo M; Solero CL; Valsecchi MG; Brain Cancer Register of the Fondazione IRCCS (Istituto Ricovero e Cura a Carattere Scientifico) Istituto Neurologico Carlo Besta
Neuro-oncology
2008
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
Journal Article
<a href="http://doi.org/10.1215/15228517-2007-038" target="_blank" rel="noreferrer">10.1215/15228517-2007-038</a>
Handgrip strength and mortality in the oldest old population: the Leiden 85-plus study
Female; Humans; Male; mortality; Follow-Up Studies; Prospective Studies; Aged; Comorbidity; Netherlands; Activities of Daily Living; Depression; Kaplan-Meier Estimate; Disability Evaluation; 80 and over; Study Design; Neuropsychological Tests; Hand Strength; Cardiovascular Diseases; Muscle Strength Dynamometer
BACKGROUND: Poor muscular strength has been shown to be associated with increased morbidity and mortality in diverse samples of middle-aged and elderly people. However, the oldest old population (i.e., over 85 years) is underrepresented in such studies. Our objective was to assess the association between muscular strength and mortality in the oldest old population. METHODS: We included 555 participants (65% women) from the Leiden 85-plus study, a prospective population-based study of all 85-year-old inhabitants of Leiden, Netherlands. We measured the handgrip strength of participants at baseline and again at age 89 years. We collected baseline data on comorbidities, functional status, levels of physical activity, and adjusted for potential confounders. During the follow-up period, we collected data on mortality. RESULTS: During a follow-up period of 9.5 years (range 8.5-10.5 years), 444 (80%) participants died. Risk for all-cause mortality was elevated among participants in the lowest tertile of handgrip strength at age 85 years (hazard ratio [HR] 1.35, 95% confidence interval [CI] 1.00-1.82, p = 0.047) and the lowest two tertiles of handgrip strength at age 89 years (HR 2.04, CI 1.24-3.35, p = 0.005 and HR 1.73, CI 1.11-2.70, p = 0.016). We also observed significantly increased mortality among participants in the tertile with the highest relative loss of handgrip strength over four years (HR 1.72, CI 1.07-2.77, p = 0.026). INTERPRETATION: Handgrip strength, a surrogate measurement of overall muscular strength, is a predictor of all-cause mortality in the oldest old population and may serve as a convenient tool for prognostication of mortality risk among elderly people.
2010-03
Ling CHY; Taekema D; de Craen Anton JM; Gussekloo J; Westendorp RG; Maier AB
Canadian Medical Association Journal
2010
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
Journal Article
<a href="http://doi.org/10.1503/cmaj.091278" target="_blank" rel="noreferrer">10.1503/cmaj.091278</a>