Philip E. Gates

Alendronate for the Treatment of Pediatric Osteogenesis Imperfecta: A Randomized Placebo-Controlled Study

CONTEXT Information on the use of oral bisphosphonate agents to treat pediatric osteogenesis imperfecta (OI) is limited. OBJECTIVE The objective of the investigation was to study the efficacy and safety of daily oral alendronate (ALN) in children with OI. DESIGN AND PARTICIPANTS We conducted a multicenter, double-blind, randomized, placebo-controlled study. One hundred thirty-nine children (aged 4-19 yr) with type I, III, or IV OI were randomized to either placebo (n = 30) or ALN (n = 109) for 2 yr. ALN doses were 5 mg/d in children less than 40 kg and 10 mg/d for those 40 kg and greater. MAIN OUTCOME MEASURES Spine areal bone mineral density (BMD) z-score, urinary N-telopeptide of collagen type I, extremity fracture incidence, vertebral area, iliac cortical width, bone pain, physical activity, and safety parameters were measured. RESULTS ALN increased spine areal BMD by 51% vs. a 12% increase with placebo (P < 0.001); the mean spine areal BMD z-score increased significantly from -4.6 to -3.3 (P < 0.001) with ALN, whereas the change in the placebo group (from -4.6 to -4.5) was insignificant. Urinary N-telopeptide of collagen type I decreased by 62% in the ALN-treated group, compared with 32% with placebo (P < 0.001). Long-bone fracture incidence, average midline vertebral height, iliac cortical width, bone pain, and physical activity were similar between groups. The incidences of clinical and laboratory adverse experiences were also similar between the treatment and placebo groups. CONCLUSIONS Oral ALN for 2 yr in pediatric patients with OI significantly decreased bone turnover and increased spine areal BMD but was not associated with improved fracture outcomes.

Function and well-being in ambulatory children with cerebral palsy

The purpose of this study was to determine whether there is a significant association between function and well-being in children with cerebral palsy. To determine this, the authors used validated measures of function (Gillette Functional Assessment Questionnaire, Gross Motor Function Classification System, Gross Motor Function Measure, and walking speed) and correlated them to health-related quality of life (HRQOL) measures (Pediatric Outcomes Data Collection Instrument, Pediatric Quality of Life instrument). In a cross-sectional study of ambulatory children with mild to moderate cerebral palsy aged 10.2 ± 3.2 years, mild to moderate decreases in function were found when compared with normative data. As the assessment of HRQOL comprises both functional well-being and psychosocial well-being, the authors decided to specify the aspect of well-being to which they were referring. It was found that the childs function was not correlated to psychosocial well-being. The children with mild cerebral palsy had greater effects on their psychosocial well-being than would be predicted by their functional disability. Functional measures were good at predicting the functional well-being but were weak at predicting the psychosocial arm of well-being.

Health-Related Quality of Life Outcomes Improve After Multilevel Surgery in Ambulatory Children With Cerebral Palsy

Background: Studies evaluating multilevel surgery to treat spastic deformity and functional deficits in cerebral palsy (CP) usually focus on data from instrumented gait analysis and clinical examination without examining functional and health-related quality of life (HRQOL) outcomes. Recently, outcome measures for well-being in children with a variety of musculoskeletal disorders have also been validated specifically for CP. Therefore, this study aimed to investigate the impact of multilevel surgery on the function and HRQOL in a group of ambulatory children with CP. Methods: In a multicenter prospective trial, 57 ambulatory children with CP, mean age 9.5 years, underwent multilevel soft tissue surgery to correct sagittal imbalance. Validated clinical outcome measures for HRQOL were administered preoperatively and postoperatively with an average follow-up time of 15.2 months. The functional and psychosocial components of the Pediatric Outcomes Data Collection Instrument (PODCI), Pediatric Quality of Life Questionnaire (PedsQL), and the Functional Assessment Questionnaire Walking Score were used. Results: Significant improvements in outcome scores occurred postoperatively in the following: PedsQL parent-total (17.6%; P < 0.001) and parent-physical sections (25.0%; P < 0.001), the Functional Assessment Questionnaire Walking Score (15.3%; P < 0.001), and the PODCI sections for transfers and basic mobility (15.8%; P < 0.001), sports and physical function (23.9%; P = 0.012), and global (12.9%; P < 0.001). Improvements also occurred in the PedsQL child-total (8.4%; P = 0.104) and child-physical sections (8.6%; P = 0.189), but these were not statistically significant. There were no significant changes in the PODCI parent-derived pain (−3.2%; P = 0.504) and happiness sections (1.9%; P = 0.645). Conclusions: Multilevel surgery in ambulatory patients with CP improves function and HRQOL. However, improved functional well-being does not imply improved psychosocial well-being, and patients and their families should be counseled accordingly. Level of Evidence: Therapeutic Level II. See Instructions to Authors for a complete description of levels of evidence.

Effects of a supported speed treadmill training exercise program on impairment and function for children with cerebral palsy

Aim  To compare the effects of a supported speed treadmill training exercise program (SSTTEP) with exercise on spasticity, strength, motor control, gait spatiotemporal parameters, gross motor skills, and physical function.

Think Globally, Act Locally: An Approach to Implementation of Clinical Practice Guidelines

BACKGROUND In an environment of concern about the rising costs of medical care, the Vermont Program for Quality in Health Care (VPQHC; the program) was incorporated in 1988 as a nonprofit organization and in 1989 was made a peer review organization by the state legislature. The program acts a resource center for health care in Vermont, coordinating three functions: implementation and maintenance of a statewide database for healthcare quality; training for health care providers in continuous quality improvement (CQI) methods and support for their CQI projects; and focusing clinical study group work on specific diagnoses or procedures. METHODS The program uses a seven-step process for implementing CQI: pick a process (modify a nationally developed guideline or develop a new guideline); select a team of people involved in doing the work; establish goals and key quality factors; document the clinical process; determine what and how to measure; measure and analyze data; and modify the process to improve. GUIDELINE: This article describes the implementation of a guideline from the American College of Obstetricians and Gynecologists (ACOG) on cesarean section (C-section). Except for a few specific contraindications, the ACOG paper states that it is reasonable to encourage vaginal birth after C-section. A related set of criteria from ACOG states that the benchmark for emergency C-section should be 30 minutes from the decision to proceed with an emergency C-section until the baby is born. CASE STUDY State C-section rates from 1985-1990 showed wide variation by hospital. Such wide variation is based as much on practice style as on sound science. C-section rates (primary and repeated) were measured, plotted, and shared with Vermont hospitals. Successful vaginal birth after C-section (VBAC) rates from the statewide data-base also were shared. Based on these data, one hospital, Hospital A, developed a plan to lower its repeated C-section rate and improve its VBAC rate. Hospital A collected and reviewed local data, which showed interesting variations. The time from decision to birth became the focus of the overall project; Hospital A designed its project to study events during the time from decision to birth. RESULTS Most recent data (1992-1993) from Hospital A shows improvement in three areas. First, 89% of patients having emergency C-sections met the goal of a 30-minute time frame from decision to incision. Second, the VBAC success rate for the same time period increased to 85.7% from 69% in the previous year, and from a mere 7% before the implementation of the project. Third, the percentage of total C-sections that were repeat C-sections fell to 36% from a high of 51%. In a follow-up evaluation, one-third of the C-section performed from October 1, 1993, to July 31, 1994 were performed because of patient refusal to attempt VBAC, suggesting that there is a communitywide culture that influences behavior. A major effort at patient education on VBAC is underway. CONCLUSIONS Think globally, act locally, might be the motto for the program. Implementing guidelines starts with obtaining national guidelines and literature but needs the use of local data to sharpen the focus on narrow areas to address. Specifically, it is unrealistic to tackle the entire problem at once. Success comes from finding specific opportunities for improvement.

Functioning and health-related quality of life of adolescents with cerebral palsy: self versus parent perspectives

Aim  To determine whether there is a difference between perspectives of functioning and health‐related quality of life (HRQL) of parents and ambulatory adolescents with spastic cerebral palsy (CP).

A prospective evaluation of the WeeFIM in patients with cerebral palsy undergoing orthopaedic surgery.

Purpose: Although frequently used in pediatric rehabilitation settings, the WeeFIM has not been tested in surgical pediatric orthopaedic patients. Methods: The WeeFIM was administered to patients with surgical cerebral palsy at defined intervals preoperatively and at both 6 and 12 months postoperatively. The age-adjusted change scores from baseline to follow-up were tested both parametrically and nonparametrically. Results: Four hundred sixty-eight patients had baseline evaluations. There were 161 six-month follow-up assessments and 108 twelve-month follow-up assessments. The baseline WeeFIM was able to separate children with different patterns of cerebral palsy. Hemiplegic patients had higher scores than diplegic and tetraplegic patients. Overall age-adjusted scores were improved at both 6 (mean increase 2.0) and 12 months (mean increase 2.2). The instrument showed significant ceiling effects for diplegic and hemiplegic patients with lower or upper extremity surgery and limited responsiveness for lower extremity surgery in tetraplegic patients. Parametrically, it showed improvements in mobility for both rhizotomy and tetraplegic upper extremity surgery. Nonparametric tests were not significant for rhizotomy mobility improvement. Conclusions: Although the WeeFIM adequately reflects the severity of neurological involvement in pediatric orthopaedic patients with cerebral palsy, it has a significant ceiling effect in diplegic and hemiplegic patients limiting responsiveness and lacks content validity for tetraplegic patients. The instrument may have some use in tetraplegic patients with upper extremity surgery and in rhizotomy patients. We recommend against its general use for orthopaedic surgery in patients with cerebral palsy lower extremity or spine surgery and in hemiplegic patients with upper extremity surgery.

Relationship between parental PODCI questionnaire and School Function Assessment in measuring performance in children with CP

Little data exists assessing the relationship between functional limitations in children with cerebral palsy (CP) and their participation in everyday activities. This prospective study evaluates the relationship between the Pediatric Outcomes Data Collection Instrument (PODCI), a functional health‐related quality of life instrument for children and their parents, and the School Function Assessment (SFA), a school‐based functional assessment. One hundred and two children with CP (80.4% dipliegia; 10.8% hemiplegia; 3.9% triplegia; 2.0% quadriplegia; 2.9% unspecified; 60 males, 42 females, mean age 11 years 8 months (SD 3y 3mo, range 6–8y), Gross Motor Function Classification System levels I to IV (13.7% Level I 50% Level II 35.3% Level III 1% Level IV), had complete PODCI and SFA assessments. Significant relationships were noted among multiple PODCI subscales and subscales of the SFA, as well as among individual questions. PODCI predicted performance in all 31 subscales of the SFA when comorbidity subscales were included (r =0.35–0.64). The PODCI in‐clinic questionnaire provides an accurate reflection of the child’s actual participation in the community setting, as assessed by the SFA. PODCI can reliably be used to help ensure that outcomes assessed in the clinic setting reflect function within the community, and can be used to help with treatment planning, goal setting, and improved patient care.

Randomized controlled trial assessing participation and quality of life in a supported speed treadmill training exercise program vs. a strengthening program for children with cerebral palsy.

OBJECTIVE A multi-site Randomized-Controlled Trial compared a home-based Supported Speed Treadmill Training Exercise Program (SSTTEP) with a strengthening exercise program in children with cerebral palsy (CP) on the following categories; Participation, quality of life (QOL), self-concept, goal attainment, and satisfaction. DESIGN Twenty-six children with spastic cerebral palsy were assigned by site-based block randomization to the SSTTEP (n=14) or strengthening exercise (n=12) group. Both groups participated in a two week clinic-based induction period and continued the intervention at home for ten weeks. Data were collected at baseline, post-intervention (12 weeks), and follow-up (16 weeks). Assessments included the Canadian Occupational Performance Measure, Childrens Assessment of Participation and Enjoyment Scale, Pediatric Quality of Life Cerebral Palsy Module, and Piers-Harris Childrens Self-Concept Scale. Evaluators were blinded to group assignment at two sites. RESULTS Satisfaction and performance on individual goals, participation, and parent-reported QOL improved in both groups with improvement maintained for four weeks post intervention. CONCLUSION The hypothesis that the SSTTEP group would have better outcomes than the exercise group was not supported. However, both groups showed that children with CP can make gains in participation, individual goals, and satisfaction following a 12-week intensive exercise intervention, and these findings persisted for four weeks post intervention.

Wound healing in orthopaedic procedures for Klippel-Trenaunay syndrome.

Klippel-Trenaunay syndrome is a triad of cutaneous hemangiomas, varicose veins, and hypertrophy of soft tissue and bone; when combined with arteriovenous fistulas, the syndrome is known as Klippel-Trenaunay-Weber syndrome. Orthopaedic surgical management of localized limb-length discrepancy or hypertrophy in these conditions is frequently indicated, especially in the lower limb. Forty orthopaedic procedures in 21 patients were retrospectively reviewed. Nine (22.5%) wound complications were identified in this study group. All the complications were associated with transverse amputations. All required significant further treatment and extension of hospital stay. Wound complications should be anticipated in patients with Klippel-Trenaunay syndrome having orthopaedic surgical procedures, especially terminal amputations.