BackgroundThe number of adolescents with chronic health conditions (CHCs) continues to increase. Medication nonadherence is a global challenge among adolescents across chronic conditions and is associated with poor health outcomes. While there has been growing interest in the use of mHealth technology to improve medication adherence among adolescents with CHCs, particularly text messaging and mobile phone apps, there has been no prior systematic review of their efficacy.ObjectiveThe purpose of this review was to systematically evaluate the most recent evidence for the efficacy of text messaging and mobile phone apps as interventions to promote medication adherence among adolescents with CHCs.MethodsPubMed, Embase, CENTRAL, PsycINFO, Web of Science, Google Scholar, and additional databases were searched from 1995 until November 2015. An additional hand search of related themes in the Journal of Medical Internet Research was also conducted. The Preferred Reporting Results of Systematic Reviews and Meta-Analyses guidelines were followed. Two reviewers independently screened titles/abstracts, assessed full-text articles, extracted data from included articles, and assessed their quality using Grades of Recommendation, Assessment, Development, and Evaluation criteria. Included studies were described in original research articles that targeted adherence in adolescents with CHCs (12-24 years-old).ResultsOf the 1423 records examined, 15 met predefined criteria: text messaging (n=12) and mobile phone apps (n=3). Most studies were performed in the United States (11/15, 73%), were randomized-controlled trials (8/15, 53%), had a sample size <50 (11/15, 73%), and included adherence self-report and/or biomarkers (9/15, 60%). Only four studies were designed based on a theoretical framework. Approaches for text messaging and mobile phone app interventions varied across studies. Seven articles (7/15, 47%) reported significant improvement in adherence with moderate to large standardized mean differences. Most of the included studies were of low or moderate quality. Studies varied in sample size, methods of adherence assessment, and definition of adherence, which prohibited performing a meta-analysis.ConclusionsThe use of text messaging and mobile phone app interventions to improve medication adherence among adolescents with CHCs has shown promising feasibility and acceptability, and there is modest evidence to support the efficacy of these interventions. Further evaluation of short- and long-term efficacy and cost-effectiveness of these interventions is warranted given the early and evolving state of the science.

Inborn errors of immunity (IEI) are a genetically heterogeneous group of disorders with a broad clinical spectrum. Identification of molecular and functional bases of these disorders is important for diagnosis, treatment and an understanding of the human immune response. We identified six unrelated males with neutropenia, infections, lymphoproliferation, humoral immune defects, and in some cases bone marrow failure associated with three different variants in the X-linked gene TLR8, encoding the endosomal Toll-like receptor 8 (TLR8). Interestingly, five patients had somatic variants in TLR8 with less than 30% mosaicism, suggesting a dominant mechanism responsible for the clinical phenotype. Mosaicism was also detected in skin-derived fibroblasts in three patients, demonstrating that mutations were not limited to the hematopoietic compartment. All patients had refractory chronic neutropenia, and three patients underwent allogeneic hematopoietic cell transplantation. All variants conferred gain-of-function to TLR8 protein, and immune phenotyping demonstrated a pro-inflammatory phenotype with activated T cells and elevated serum cytokines associated with impaired B cell maturation. Differentiation of myeloid cells from patient-derived induce pluripotent stem cells demonstrated increased responsiveness to TLR8. Together these findings demonstrate that gain-of-function variants in TLR8 lead to a novel childhood-onset IEI with lymphoproliferation, neutropenia, infectious susceptibility, B and T cell defects, and in some cases bone marrow failure. Somatic mosaicism is a prominent molecular mechanism of this new disease.

Prior to the 1950s, survival from Wilms tumor (WT) was less than 10%. Today, a child diagnosed with WT has a greater than 90% chance of survival. These gains in survival rates from WT are attributed largely to improvements in multimodal therapy: Enhanced surgical techniques leading to decreased operative mortality, optimization of more effective chemotherapy regimens (specifically, dactinomycin and vincristine), and inclusion of radiation therapy in treatment protocols. More recent improvements in survival, however, can be attributed to a growing understanding of the molecular landscape of Wilms tumor. Particularly, identification of biologic markers portending poor prognosis has facilitated risk stratification to tailor therapy that achieves the best possible outcome with the least possible toxicity. The aim of this review is to (1) outline the specific biologic markers that have been associated with prognosis in WT and (2) provide an overview of the current use of biologic and other factors to stratify risk and assign treatment accordingly.

Purpose of review Graft-versus-host disease (GVHD) following hematopoietic cell transplant (HCT) has a significant impact on morbidity and mortality among recipients. Predicting the long-term outcomes at the time of diagnosis of GVHD or even after response to up-front therapy can be challenging and only has modest accuracy. With biomarkers available to help guide decision-making, the landscape of GVHD is evolving. Recent findings Several acute GVHD biomarkers have been identified, with some better able to categorize patients based on their GVHD severity and potential for refractory disease than standard clinical staging or response criteria. Summary Biomarkers are now being incorporated into the clinical trial design for both high and low-risk GVHD. These findings will likely impact how clinical care is delivered in the future as improved risk stratification has the potential to improve outcomes by providing individualized treatment plans for affected patients.