Severe ROP is associated with maturational delay in the optic radiations, PLIC, external capsule and posterior white matter, housing the primary visual and motor pathways, and is associated with poorer cognitive and motor outcomes at 18 months' CA.
Incidence and time of onset of germinal matrix/intraventricular hemorrhage (GM/IVH) were prospectively ascertained in 1,105 infants weighing < or = 2,000 g at birth, a cohort comprising about 85% of all births of that weight born from September 1984 to June 1987 in the central New Jersey counties of Ocean, Monmouth, and Middlesex. Cranial ultrasonography was performed as nearly as possible to age 4 hours, 24 hours, and 7 days. Each scan was reviewed by two independent readers and, if necessary, a third; consensus was achieved on scan of first diagnosis of GM/IVH in 965 of the 1,079 infants with assessable scans. The cumulative incidence of GM/IVH in the first week of life was 24.6% (265/1,079). In the 965 infants with consensus diagnoses, the first scan, at 4.9 +/- 2.2 hours, yielded the highest incidence--10.6% (95/899). Incidence by the second scan (25.1 +/- 4.9 hours) was 6.0% (49/813), and by the third scan (7.2 +/- 0.8 days), 9.0% (64/715). The iterative algorithm for interval-censored data developed by Turnbull (J R Stat Soc [B] 1976;8:290-5) was used to estimate the most likely time of onset based on time of first diagnosis. From 34% to 44% of hemorrhages were present at the first opportunity to scan, which in these data was at age 1 hour. At least a third of GM/IVH in infants < or = 2,000 g appears to be of congenital or immediate postnatal onset.
Purpose
To describe a series of children with extensive PNF or treatment refractory PLGG treated on a compassionate basis with trametinib.
Methods
We report on six patients with NF‐1 treated with trametinib on a compassionate basis at British Columbia Children's Hospital since 2017. Data were collected retrospectively from the patient record. RAPNO and volumetric criteria were used to evaluate the response of intracranial and extracranial lesions, respectively.
Results
Subjects were 21 months to 14 years old at the time of initiation of trametinib therapy and 3/6 subjects are male. Duration of therapy was 4–28 months at the time of this report. All patients had partial response or were stable on analysis. Two patients with life‐threatening PNF had a partial radiographic response in tandem with significant clinical improvement and developmental catch up. One subject discontinued therapy after 6 months due to paronychia and inadequate response. The most common adverse effect (AE) was grade 1–2 paronychia or dermatitis in 5/6 patients. There were no grade 3 or 4 AEs. At the time of this report, five patients remain on therapy.
Conclusion
Trametinib is an effective therapy for advanced PNF and refractory PLGG in patients with NF‐1 and is well tolerated in children. Further data and clinical trials are required to assess tolerance, efficacy and durability of response, and length of treatment required in such patients.
Background:
The Centers for Medicare and Medicaid Services have proposed 30-day ischemic stroke risk-standardized mortality rates that include adjustment for stroke severity using the National Institute of Health Stroke Scale (NIHSS), which is often undocumented. We used simulations to quantify the effect of missing NIHSS data on the accuracy of hospital-level ischemic stroke risk-standardized mortality rate profiling for 100 hypothetical hospitals with different case volumes.
Methods and Results:
We generated simulated data sets of patients with NIHSS scores and other predictors of 30-day mortality based on empirical analysis of data from 7654 patients with ischemic stroke in the Michigan Stroke Registry. We assigned and rank-ordered a true (known) 30-day mortality rate to each hospital in the simulated data sets of N=100 hospitals with either low (100 patients with stroke), medium (300), or high (500) case volumes. We then estimated and rank-ordered 30-day risk-standardized mortality rates for the N=100 hospitals in each simulated data set using hierarchical logistic regression models. In each data set, we systematically varied the rate of missing NIHSS data and whether missing NIHSS data was independent (missing completely at random) or dependent (missing not at random) on the NIHSS score. With no missing NIHSS data, the Spearman correlation between the true hospital performance rank order assigned during data set generation and the estimated 30-day risk-standardized mortality rate rank order was 0.72, 0.88, and 0.91 in low, medium, and high volume hospitals, respectively. However, this fell to as low as 0.50, 0.71, and 0.79 as missing NIHSS data reached 70%.
Conclusions:
Missing NIHSS data had substantial detrimental effects on the accuracy of profiling of ischemic stroke mortality, especially in lower volume hospitals. Even with complete NIHSS documentation, significant limitations in ischemic stroke mortality profiling remain.
We report a prospective study of the feasibility of employing specially trained physiotherapists to screen neonates for congenital dislocation of the hip. During ten years 42,241 babies were screened, using clinical tests; 255 were diagnosed and treated by a Pavlik harness. In the same period 13 children presented late with congenital dislocation of the hip which had not been detected by the screening programme.
Assessment of visual function by health workers may be a valuable tool in improving surgical uptake by encouraging both health personnel and patients to recognise that they have diYculties undertaking activities of daily living as well as a measure of monitoring and evaluating cataract outcomes. (Br J Ophthalmol 1999;83:792-795)
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