Shared Decision-Making and Children With Disabilities: Pathways to Consensus.

Shared decision-making (SDM) promotes family and clinician collaboration, with ultimate goals of improved health and satisfaction. This clinical report provides a basis for a systematic approach to the implementation of SDM by clinicians for children with disabilities. Often in the discussion of treatment plans, there are gaps between the child's/family's values, priorities, and understanding of perceived "best choices" and those of the clinician. When conducted well, SDM affords an appropriate balance incorporating voices of all stakeholders, ultimately supporting both the child/family and clinician. With increasing knowledge of and functional use of SDM skills, the clinician will become an effective partner in the decision-making process with families, providing family-centered care. The outcome of the process will support the beneficence of the physician, the authority of the family, and the autonomy and well-being of the child.

Nonoral feeding for children and youth with developmental or acquired disabilities.

The decision to initiate enteral feedings is multifaceted, involving medical, financial, cultural, and emotional considerations. Children who have developmental or acquired disabilities are at risk for having primary and secondary conditions that affect growth and nutritional well-being. This clinical report provides (1) an overview of clinical issues in children who have developmental or acquired disabilities that may prompt a need to consider nonoral feedings, (2) a systematic way to support the child and family in clinical decisions related to initiating nonoral feeding, (3) information on surgical options that the family may need to consider in that decision-making process, and (4) pediatric guidance for ongoing care after initiation of nonoral feeding intervention, including care of the gastrostomy tube and skin site. Ongoing medical and psychosocial support is needed after initiation of nonoral feedings and is best provided through the collaborative efforts of the family and a team of professionals that may include the pediatrician, dietitian, social worker, and/or therapists.

Early intervention, IDEA Part C services, and the medical home: collaboration for best practice and best outcomes.

The medical home and the Individuals With Disabilities Education Act Part C Early Intervention Program share many common purposes for infants and children ages 0 to 3 years, not the least of which is a family-centered focus. Professionals in pediatric medical home practices see substantial numbers of infants and toddlers with developmental delays and/or complex chronic conditions. Economic, health, and family-focused data each underscore the critical role of timely referral for relationship-based, individualized, accessible early intervention services and the need for collaborative partnerships in care. The medical home process and Individuals With Disabilities Education Act Part C policy both support nurturing relationships and family-centered care; both offer clear value in terms of economic and health outcomes. Best practice models for early intervention services incorporate learning in the natural environment and coaching models. Proactive medical homes provide strategies for effective developmental surveillance, family-centered resources, and tools to support high-risk groups, and comanagement of infants with special health care needs, including the monitoring of services provided and outcomes achieved.

Fetal spine findings on MRI and associated outcomes in children with open neural tube defects.

OBJECTIVE: The purpose of this study is to determine the relationship between abnormal fetal spine findings on MRI and adverse outcomes in children with open neural tube defects. MATERIALS AND METHODS: This was a review of pregnancies complicated by myelomeningocele referred for fetal MRI from 2001 to 2007 and followed postnatally at a spina bifida treatment center. MRI scans were reviewed to determine lesion level (T-L2, L3-4, or L5-S), interpediculate distance (≤ 10 or > 10 mm), vertebral segment span, and presence or absence of covering membrane. Ambulation was assessed in children 3 years old or older. Bladder dysfunction was termed as high-risk if renal damage was present or if urodynamic studies indicated increased risk for renal damage. Statistical analyses included chi-square, Mantel-Haenszel test for trend, and logistic regression. RESULTS: MRI was performed in 36 pregnancies with fetal myelomeningocele at a mean (± SD) of 27 ± 6 weeks, with subsequent delivery at 38 ± 1 week. Outcomes were assessed at 3.2 years (range, 2.4-5.1 years), and 23 children were 3 years old or older. Higher lesion level was associated with dysphagia: T-L2, 50%; L3-4, 45%; and L5-S, 13% (p < 0.05). The absence of covering membrane was associated with scoliosis (36% vs 0% with membrane present) and with high-risk bladder dysfunction (71% vs 36%; both p < 0.05). Higher lesion level, larger segment span, and interpediculate distance greater than 10 mm were associated with full-time wheelchair use (all p < 0.05). CONCLUSION: In fetuses with myelomeningoceles, higher and larger lesions on MRI were significantly associated with full-time wheelchair use. High lesion level was associated with dysphagia. The absence of a covering membrane was associated with scoliosis and high-risk bladder dysfunction.

Central nervous system findings on fetal magnetic resonance imaging and outcomes in children with spina bifida.

OBJECTIVE: To estimate the relationship between fetal magnetic resonance imaging (MRI) findings of ventriculomegaly, cerebellar herniation, extraaxial space effacement and adverse outcomes in children with spina bifida. METHODS: This was a review of all pregnancies with spina bifida referred for fetal MRI from 2001 to 2007 and cared for postnatally at our spina bifida referral center. Degree of cerebellar herniation was based on lowest cervical vertebral level reached. Extraaxial space was considered effaced if too small to be measured above or below the tentorium cerebelli. Bladder dysfunction was termed high risk if renal damage was present or urodynamic studies indicated increased risk for renal damage. Ambulation was assessed in children age 3 years or older. Statistical analyses included chi square, Mantel-Haenszel test, and logistic regression. RESULTS: Magnetic resonance imaging was performed in 36 pregnancies with fetal spina bifida at 27+/-6 weeks with subsequent delivery at 38+/-1 weeks. Outcomes were assessed at 3.2 years (range 2.4-5.1); 23 children were age 3 years or older. If the cerebellum was above the foramen magnum or had herniated to C2, C3, or C4, respectively, childhood seizures occurred in 0%, 7%, 21%, and 100%; high-risk bladder dysfunction in 33%, 33%, 71%, and 100%; and inability to ambulate independently in 20%, 70%, and 100% (no C4 cases) all P<.05. Ventriculomegaly, cerebellar herniation, and extraaxial space effacement were significantly associated with the need for ventriculoperitoneal shunt; however, 94% of children required shunt placement. CONCLUSION: In fetuses with spina bifida, worsening cerebellar herniation on MRI was significantly associated with childhood seizure activity, high-risk bladder dysfunction, and lack of independent ambulation. LEVEL OF EVIDENCE: II.

Incidence of new onset metabolic acidosis following enteroplasty for myelomeningocele.

PURPOSE: Extant literature is mixed regarding risk of metabolic acidosis after enteroplasty for myelomeningocele. This study is the first known attempt to describe the pattern of developing metabolic acidosis in a group of children who underwent enteroplasty and served as their own controls. Multiple preoperative and postoperative laboratory measures for each child were obtained for comparison. MATERIALS AND METHODS: This retrospective cohort study allowed participants to serve as their own controls for pre-intervention and post-intervention analysis. The setting was a tertiary, university affiliated, interdisciplinary spina bifida program. All patients followed in the spina bifida program who had undergone ileal or colonic enteroplasty were included for review (total 113). Strict exclusion criteria were preoperatively diagnosed renal insufficiency, preexisting metabolic acidosis consistent with renal tubular acidosis (pH less than 7.35, bicarbonate 20 mmol/l or less) and history of augmentation using gastric or ureteral tissue. Final analysis included 71 children who met inclusion criteria. Children in our spina bifida program periodically undergo routine laboratory evaluation of electrolytes, blood urea nitrogen, creatinine, blood count, and venous blood gases including pH, bicarbonate and partial pressure of carbon dioxide. Primary outcome measures were comparative shifts in blood gases and electrolytes that would confirm the new onset of metabolic acidosis after enteroplasty. Changes in electrolytes and serum creatinine were secondary outcome measures to identify potential markers for postoperative effects. With each child as his/her own control, analysis included paired t tests. RESULTS: No statistically significant differences (p <0.05) were found when comparing laboratory values before and after bladder augmentation, including pH, bicarbonate, partial pressure of carbon dioxide and electrolytes. No child had metabolic acidosis based on the aforementioned criteria. Followup ranged from 1 to 138 months after enteroplasty (mean 46.8). Respiratory compensation was considered in the analysis, and no difference in partial pressure of carbon dioxide following surgery was noted (p = 0.65). CONCLUSIONS: To our knowledge no previous study has examined the matched paired results of before and after development of metabolic acidosis among children (serving as their own controls) with myelomeningocele undergoing ileal or colonic enteroplasty. The negative statistical results in this controlled cohort are clinically significant. If a child with myelomeningocele has metabolic acidosis after enteroplasty, other clinical reasons beyond the effects of surgery warrant careful consideration.

Limbic tract anomalies in pediatric myelomeningocele and Chiari II malformation: anatomic correlations with memory and learning--initial investigation.

PURPOSE: To prospectively determine anomalies of limbic tracts and to describe the relationship between these anomalies, seen on diffusion-tensor magnetic resonance (MR) and fiber tract (FT) reconstruction images, and learning and memory in children with myelomeningocele (MM) and Chiari II malformation. MATERIALS AND METHODS: The investigation was HIPAA compliant and approved by institutional review boards; informed consent was obtained. In seven male and six female patients (aged 6 months to 16 years) with MM and Chiari II malformation, diffusion-tensor imaging and FT reconstruction were performed. FT reconstruction was generated with fractional anisotropy continuous tracking algorithm and manually drawn regions of interest. Limbic tract abnormalities were assessed on FT reconstruction images by an experienced pediatric neuroradiologist blinded to results of cognitive testing. Nine patients met criteria for memory and learning testing by a trained cognitive neuroscientist blinded to MR results. Exact Wilcoxon rank sum test was used to compare performance with learning and memory tasks in two groups. RESULTS: Eleven of 13 patients had defects within fornices and/or cingulum; three patients had aberrant fibers of cingulum. In nine patients, six had deficits in general memory; four, in learning; and four, in both. Atresia or hypoplasia of crura and body of fornices was noted in six patients with memory deficits and four patients with learning deficits. Five of six patients with memory deficits and three of four with learning deficits had hypoplasia or atresia of cingulum. Exact Wilcoxon rank sum test demonstrated significantly poorer performance for nonverbal immediate recall tasks in patients with anomalies of the fornix compared with those without (P = .04, exact two-tailed test). CONCLUSION: Diffusion-tensor and FT reconstruction images revealed that limbic fiber abnormalities were common in patients with MM and Chiari II malformation. Nonverbal immediate recall task performance appeared to be related to abnormalities of the fornix.

Memory and selective learning in children with spina bifida-myelomeningocele and shunted hydrocephalus: a preliminary study.

BACKGROUND: Selective learning is the ability to select items of relevance from among less important items. Limited evidence exists regarding the efficiency with which children with spina bifida-myelomeningocele and shunted hydrocephalus (SB/SH) are able to learn information. This report describes initial data related to components of learning and metacognitive skills in children with SB/SH. METHODS: Twenty six children with SB/SH and 26 controls (age: 7-16 y) with average intelligence, and monolingual English-speaking backgrounds participated in the study. Exclusion criteria for the SB/SH group were: prior history of shunt infection, history of seizure or shunt malfunction within the previous three months, prior diagnoses of attention disorders and/or clinical depression. Children were presented lists of words with equal exemplars each of two distinct semantic categories (e.g. fruits, animals), and told to make as high a score as possible by learning the words. The value of the words was designated by category membership (e.g. animals = low value; fruits = high value). The total number of words learned across three learning trials was used to determine memory span. Selective learning efficiency (SLE) was computed as the efficiency with which items of greater value were selectively learned across three trials. RESULTS: Children with SB/SH did worse than controls on memory span (P < 0.05). Although SLE was not significantly different between groups, when asked what strategy was used in the selective learning tasks, 65% of the SB/SH children said they tried to remember all words (inefficient strategy). In contrast, 85% of controls said they tried to remember the higher value words--the more efficient strategy. CONCLUSION: Success in school is often dependent on the ability to recall important facts selectively and ignore less important information. Children with SB/SH in our study had a poor memory span and were unable to monitor and report an efficient and workable metacognitive strategy required to remember a list of words. Preliminary findings may begin to explain our previous clinical and research findings wherein children with SB/SH often focus on extraneous details, but demonstrate difficulty remembering the main gist of a story/event.

Efficacy and safety of oxybutynin in children with detrusor hyperreflexia secondary to neurogenic bladder dysfunction.

PURPOSE: We evaluated the efficacy and safety of oxybutynin in children with detrusor hyperreflexia due to neurological conditions. MATERIALS AND METHODS: Study 1--A prospective, open label trial of 3 formulations of oxybutynin (tablets, syrup and extended release tablets) was conducted for 24 weeks in children 6 to 15 years old with detrusor hyperreflexia who used oxybutynin and clean intermittent catheterization. The effect of treatment on average urine volume per catheterization and on secondary urodynamic outcomes was evaluated. Study 2--The efficacy and safety of oxybutynin syrup were evaluated urodynamically in an open label study of children 1 to 5 years old with detrusor hyperreflexia who used oxybutynin and clean intermittent catheterization. RESULTS: Study 1--Mean urine volume per catheterization (+/- SEM) increased by 25.5 +/- 5.9 ml (p <0.001). Maximal cystometric capacity increased by 75.4 +/- 9.8 ml (p <0.001). Mean detrusor and intravesical pressures were significantly decreased by -9.2 +/- 2.3 (p < or =0.001) and -7.5 +/- 2.5 cm H2O (p <0.004), respectively, at week 24. Of 61 children with uninhibited detrusor contractions 15 cm H2O or greater at baseline 34 did not have them at week 24 (p <0.001). Improvements in bladder function were consistent across all oxybutynin formulations. Study 2--Mean maximal cystometric capacity increased significantly by 71.5 +/- 21.99 ml (p = 0.005). At study end only 12.5% of patients had uninhibited detrusor contractions 15 cm H2O or greater compared with 68.8% at baseline (p = 0.004). Oxybutynin was well tolerated in both studies. There were no serious treatment related adverse events. CONCLUSIONS: All 3 formulations of oxybutynin are safe and effective in children with neurogenic bladder dysfunction.

Regulatory considerations of pharmaceutical solid polymorphism in Abbreviated New Drug Applications (ANDAs).

A sponsor of an Abbreviated New Drug Application (ANDA) must have information to show that the proposed generic product and the innovator product are both pharmaceutically equivalent and bioequivalent, and therefore, therapeutically equivalent. Many pharmaceutical solids exist in several crystalline forms and thus exhibit polymorphism. Polymorphism may result in differences in the physico-chemical properties of the active ingredient and variations in these properties may render a generic drug product to be bioinequivalent to the innovator brand. For this reason, in ANDAs, careful attention is paid to the effect of polymorphism in the context of generic drug product equivalency. This review discusses the impact of polymorphism on drug product manufacturability, quality, and performance. Conclusions from this analysis demonstrate that pharmaceutical solid polymorphism has no relevance to the determination of drug substance "sameness" in ANDAs. Three decision trees for solid oral dosage forms or liquid suspensions are provided for evaluating when and how polymorphs of drug substances should be monitored and controlled in ANDA submissions. Case studies from ANDAs are provided which demonstrate the irrelevance of polymorphism to the determination of drug substance "sameness". These case studies also illustrate the conceptual framework from these decision trees and illustrate how their general principles are sufficient to assure both the quality and the therapeutic equivalence of marketed generic drug products.

A temperament for learning: The limbic system and myelomeningocele.

: This article was the winner of the triennial Casey Holter Memorial Prize awarded by the Society for Research into Hydrocephalus and Spina Bifida, 2004. ABSTRACT: This essay explores the link between the limbic/hypothalamic systems within the complex conditions of hydrocephalus and myelomeningocele. Acknowledging the neuroanatomical and neuroendocrine risks inherent in the developing brains of these individuals, we focus on the converging components of temperament, cognition, and language.

Scientific considerations of pharmaceutical solid polymorphism in abbreviated new drug applications.

PURPOSE: This commentary is intended to provide a scientific perspective on pharmaceutical solid polymorphism in Abbreviated New Drug Applications (ANDAs). METHODS: This report proposes recommendations for monitoring and controlling drug substance polymorphs and describes scientific considerations of pharmaceutical solid polymorphism in the determination of drug substance sameness. RESULTS: It presents three decision trees for solid oral dosage forms or liquids containing undissolved drug substances to provide a process for evaluating when and how polymorphs of drug substances are monitored and controlled in ANDA submissions. CONCLUSIONS: It is scientifically concluded that differences in polymorphic composition of drug substances in generic drug products and reference-listed drugs are not directly relevant in the determination of drug substance sameness in ANDAs.