New stimulant formulations for pediatric attention-deficit/hyperactivity disorder: a case-based approach for the primary care provider.

PURPOSE OF REVIEW: To provide an up-to-date clinical review of U.S. Food and Drug Administration (FDA)-approved stimulant medications for attention-deficit/hyperactivity disorder (ADHD), including a framework for individualized treatment by primary care pediatric providers. RECENT FINDINGS: Stimulant medications are first-line agents for pediatric ADHD. Since 2012, 11 novel stimulant medications have been approved by the FDA for the treatment of ADHD. Because of an expanded formulary of available methylphenidate-based and amphetamine-based stimulants, primary care providers may be unfamiliar with some novel medications outside a select formulary. SUMMARY: The current broad formulary of methylphenidate-based and amphetamine-based stimulants provides primary care clinicians with a greater opportunity for personalized medicine within the patient-centered medical home. Through a systematic review of prior relevant medication trials, a consideration of daily symptom burden and thoughtful pragmatics, primary care providers can offer a more precise, customized stimulant treatment.

Evaluation of Community Programs for Early Childhood Development: Parental Perspectives and Recommendations.

Objectives Optimal early childhood development is crucial for promoting positive child health outcomes. Community programs supporting child development are available throughout the United States but general parental perceptions of such programs are not well understood. This study aimed to examine parental perceptions of community programs for early childhood development in a semi-urban city of the US. Methods Data were collected from focus groups (n = 4) composed of English-fluent parents from the local community with at least one child aged 0-5 years. After generation of verbatim transcripts, data were analyzed by two independent coders in order to identify themes. Results Parental perceptions were categorized into four areas: (1) Utilization of community services, (2) Helpful aspects of community services, (3) Negative aspects of community services and (4) Parental recommendations for improved resource utilization. Helpful aspects identified included social and economic support, provision of parental education, and developmental screening and medical support. Negative aspects included utilization of standardized assessment tools, awareness of agencies and resources, and access to services. In order to improve resource utilization, parents suggested improved communication with parents and the child's medical home, transparency, and translation of program information into other languages. Conclusions For Practice Overall, participants felt that community programs that support early childhood development and parenting were helpful. However, community agencies can improve on communication with parents and medical providers as well as translation of program information.

A Severe Case of Congenital Thrombotic Thrombocytopenia Purpura Resulting From Compound Heterozygosity Involving a Novel ADAMTS13 Pathogenic Variant.

We report a 9-year-old Chinese girl with congenital thrombotic thrombocytopenic purpura found to be a compound heterozygote for 2 pathogenic variants in the ADAMTS13 gene, including a novel variation. The girl suffered from recurrent, life-threatening episodes of thrombocytopenia and hemolysis, and laboratory testing showed ADAMST13 enzyme activity of <5%. Sequencing of the ADAMTS13 gene revealed a previously reported missense variant, c.1787C>T (p.Ala596Val), and a novel duplication defined as c.1007_1025dup19 (p.Asp343Leufs*53); the duplication is predicted to result in a premature stop codon and protein truncation. We propose that this novel variant is partly responsible for the patient's early-onset and severe phenotype.

Elective cholecystectomy reduces morbidity of cholelithiasis in pediatric sickle cell disease.

BACKGROUND: Cholelithiasis is a frequent complication in pediatric sickle cell disease (SCD). Though it is standard practice to perform a cholecystectomy in pediatric SCD patients with symptoms of cholelithiasis, the use of elective cholecystectomy for asymptomatic patients remains controversial. PROCEDURE: Records of 191 pediatric sickle cell patients with cholelithiasis who underwent cholecystectomy were retrospectively reviewed. Patients classified as follows: (i) elective-no preoperative symptoms, cholelithiasis on screening ultrasound, comprehensive preoperative plan; (ii) symptomatic-preoperative symptoms of cholelithiasis on diagnostic ultrasound, comprehensive preoperative plan; or (iii) emergent-hospitalization for acute cholecystitis symptoms, cholelithiasis on diagnostic ultrasound, limited preoperative preparation. We compared the morbidity of cholecystectomy by examining pre- and post-cholecystectomy hospital admission days, length of stay for cholecystectomy, and surgical complications. RESULTS: Patients with SCD underwent a total of 191 cholecystectomies over a 10-year period: 51 elective, 110 symptomatic, and 30 emergent. Patients who required emergent cholecystectomy had a longer postoperative hospitalization time than elective or symptomatic cholecystectomy (7.3 vs 4.3, P < 0.001). Baseline values for total bilirubin and aspartate aminotransferase (AST) were significantly elevated (P < 0.02 and P < 0.07, respectively) in patients requiring emergent cholecystectomy. CONCLUSIONS: This represents the largest reported retrospective review of pediatric cholelithiasis and cholecystectomy in SCD to date. These data strongly suggest that elective cholecystectomy decreases morbidity associated with emergent cholecystectomy. The overall outcomes for symptomatic and elective patients are favorable. However, our study indicates the need for prospective studies to identify clinical indicators for those emergent patients.