Longitudinal changes in clinical outcome measures in COL6-related dystrophies and LAMA2-related dystrophies.

OBJECTIVE: To identify the rate of change of clinical outcome measures in children with 2 types of congenital muscular dystrophy (CMD), COL6-related dystrophies (COL6-RDs) and LAMA2-related dystrophies (LAMA2-RDs). METHODS: Over the course of 4 years, 47 individuals (23 with COL6-RD and 24 with LAMA2-RD) 4 to 22 years of age were evaluated. Assessments included the Motor Function Measure 32 (MFM32), myometry (knee flexors and extensors, elbow flexors and extensors), goniometry (knee and elbow extension), pulmonary function tests, and quality-of-life measures. Separate linear mixed-effects models were fitted for each outcome measurement, with subject-specific random intercepts. RESULTS: Total MFM32 scores for COL6-RDs and LAMA2-RDs decreased at a rate of 4.01 and 2.60 points, respectively, each year (p < 0.01). All muscle groups except elbow flexors for individuals with COL6-RDs decreased in strength between 1.70% (p < 0.05) and 2.55% (p < 0.01). Range-of-motion measurements decreased by 3.21° (p < 0.05) at the left elbow each year in individuals with LAMA2-RDs and 2.35° (p < 0.01) in right knee extension each year in individuals with COL6-RDs. Pulmonary function demonstrated a yearly decline in sitting forced vital capacity percent predicted of 3.03% (p < 0.01) in individuals with COL6-RDs. There was no significant change in quality-of-life measures analyzed. CONCLUSION: Results of this study describe the rate of change of motor function as measured by the MFM32, muscle strength, range of motion, and pulmonary function in individuals with COL6-RDs and LAMA2-RDs.

Prevalence, Distribution, and Risk Factors Associated With Macracanthorhynchus ingens Infections In Raccoons From Ontario, Canada.

Macracanthorhynchus ingens is an acanthocephalan parasite commonly found in raccoons ( Procyon lotor) in the United States. Little is known, however, about the prevalence and distribution of M. ingens in raccoons in Canada. Our objective was to investigate the prevalence, distribution, and risk factors associated with M. ingens infection in raccoons in southern Ontario, Canada. Raccoon carcasses submitted to the Ontario/Nunavut region of the Canadian Wildlife Health Cooperative for post mortem from June 2016 to January 2017 (n = 380) were examined for the presence of gastrointestinal helminths. Macracanthorhynchus ingens was found in raccoons from areas across southern Ontario where carcasses were submitted. The prevalence of M. ingens in our sample was 14.0% (95% CI = 10.6-17.8) with a median of 4 worms per infected host (range 1-46). Univariable logistic regression modeling was conducted to examine the influence of age, sex, season, degree of urbanization (urban/suburban/rural), and Baylisascaris procyonis infection on the presence of M. ingens. Significant associations were found between M. ingens infection and degree of urbanization as well as B. procyonis infection. No associations were found between M. ingens infection and age, sex, or season. To our knowledge, this is the first report describing the prevalence and distribution of M. ingens in raccoons in Canada.

Anophthalmia in a Wild Eastern Gray Squirrel (Sciurus carolinensis).

We describe bilateral true anophthalmia in a juvenile female eastern gray squirrel (Sciurus carolinensis) with histologic confirmation that orbital contents lacked ocular tissues. Additionally, the optic chiasm of the brain was absent and axon density in the optic tract adjacent to the lateral geniculate nucleus was reduced.

Results of a two-year pilot study of clinical outcome measures in collagen VI- and laminin alpha2-related congenital muscular dystrophies.

Potential therapies are currently under development for two congenital muscular dystrophy (CMD) subtypes: collagen VI-related muscular dystrophy (COL6-RD) and laminin alpha 2-related dystrophy (LAMA2-RD). However, appropriate clinical outcome measures to be used in clinical trials have not been validated in CMDs. We conducted a two-year pilot study to evaluate feasibility, reliability, and validity of various outcome measures, particularly the Motor Function Measure 32, in 33 subjects with COL6-RD and LAMA2-RD. In the first year, outcome measures tested included: Motor Function Measure 32 (MFM32), forced vital capacity (FVC) percent predicted sitting, myometry, goniometry, 10-meter walk, Egen Klassification 2, and PedsQL(TM) Generic and Neuromuscular Cores. In the second year, we added the North Star Ambulatory Assessment (NSAA), Hammersmith Functional Motor Scale (HFMS), timed functional tests, Measure of Activity Limitations (ACTIVLIM), Quality of Upper Extremity Skills Test (QUEST), and Patient-Reported Outcomes Measurement Information System (PROMIS) fatigue subscale. The MFM32 showed strong inter-rater (0.92) and internal consistency (0.96) reliabilities. Concurrent validity for the MFM32 was supported by large correlations (range 0.623-0.936) with the following: FVC, NSAA, HFMS, timed functional tests, ACTIVLIM, and QUEST. Significant correlations of the MFM32 were also found with select myometry measurements, mainly of the proximal extremities and domains of the PedsQL(TM) scales focusing on physical health and neuromuscular disease. Goniometry measurements were less reliable. The Motor Function Measure is reliable and valid in the two specific subtypes of CMD evaluated, COL6-RD and LAMA2-RD. The NSAA is useful as a complementary outcome measure in ambulatory individuals. Preliminary concurrent validity of several other clinical outcome measures was also demonstrated for these subtypes.

Long-term survival and quality of life in cardiac surgical patients with prolonged intensive care unit length of stay.

BACKGROUND: Patients with prolonged intensive care unit (ICU) stays after cardiac operations are labor intensive and expensive. We sought to determine whether exhaustive ICU efforts result in survival or quality-of-life benefits and whether outcome could be predicted. METHODS: We retrospectively analyzed all adult cardiac surgical patients in 1998 for ICU stays more than 14 days. Data were analyzed to create multiple organ dysfunction scores (MODS, range 0 to 24) and hospital charges. Follow-up was conducted 1 and 2 years apart for survival and quality-of-life evaluation. RESULTS: Forty-nine patients remained in the ICU more than 14 days, comprising 3.8% of our patients but 28% of total ICU bed time. This population had a 28.5% hospital mortality, which was greater than those in the ICU less than 14 days (5.3%, p < 0.05). By 2 years, 22 of the 35 discharged patients were alive, 16 of whom had a normal quality of life. Patients alive at 2 years had lower MODS at day 14 than those who died (2.6 +/- 1.4 versus 5.5 +/- 3.8; p < 0.005) as well as lower hospital costs ($223,000 +/- $128,000 versus $306,000 +/- $128,000; p < 0.05). No patient with an MODS of at least 6 at day 14 survived. CONCLUSIONS: Patients remaining in the ICU for more than 14 days suffer a higher mortality at greater expense. A MODS at day 14 may help predict those who will not enjoy long-term survival and thus aid in the decision to terminate care.

Three-year follow-up of a randomized trial comparing focal/grid photocoagulation and intravitreal triamcinolone for diabetic macular edema.

OBJECTIVE: To report 3-year outcomes of patients who participated in a randomized trial evaluating 1-mg and 4-mg doses of preservative-free intravitreal triamcinolone compared with focal/grid photocoagulation for treatment of diabetic macular edema. METHODS: Eyes with diabetic macular edema and visual acuities of 20/40 to 20/320 were randomly assigned to focal/grid photocoagulation or 1 mg or 4 mg of triamcinolone. At the conclusion of the trial, 3-year follow-up data were available in 306 eyes. RESULTS: Between 2 years (time of the primary outcome) and 3 years, more eyes improved than worsened in all 3 treatment groups. Change in visual acuity letter score from baseline to 3 years was +5 in the laser group and 0 in each triamcinolone group. The cumulative probability of cataract surgery by 3 years was 31%, 46%, and 83% in the laser and 1-mg and 4-mg triamcinolone groups, respectively. Intraocular pressure increased by more than 10 mm Hg at any visit in 4%, 18%, and 33% of eyes, respectively. CONCLUSIONS: Results in a subset of randomized subjects who completed the 3-year follow-up are consistent with previously published 2-year results and do not indicate a long-term benefit of intravitreal triamcinolone relative to focal/grid photocoagulation in patients with diabetic macular edema similar to those studied in this clinical trial. Most eyes receiving 4 mg of triamcinolone as given in this study are likely to require cataract surgery. TRIAL REGISTRATION: clinicaltrials.gov Identifier: NCT00367133.