Adolescent Patients Research Articles

Segmental deformity markers offer novel indicators of deformity progression risk in deformity-matched adolescent idiopathic scoliosis patients.

Identification of adolescent idiopathic scoliosis (AIS) patients with mild curvatures who pose significant risk of progressing to severe levels of curvatures is of paramount importance for clinical care. This study aimed to compare segmental deformity changes in AIS sub-cohorts that are dichotomised by progression status. Thirty-six female participants with Lenke 1 AIS curves were investigated with sequential MRIs during growth. Scans were reformatted to measure orthogonal segmental parameters, including sagittal/coronal wedging angles and axial rotation angles. Participants were dichotomised by progression. Two-tailed, independent sample t-tests were used to compare sub-cohort multi-segmental and segmental deformity parameters. Measurements were compared at each scan number and variable rates of change were determined using actual time between measures. AIS progression status sub-cohorts were comparable at scan 1 for multi-segmental deformity parameters (e.g. major thoracic curve angle, rib hump, kyphosis) (P > 0.05). However, apical measures of coronal IVD wedging, axial IVD rotation and axial vertebral rotation were segmental parameters at scan 1 which were larger for participants whose AIS would later go on to clinically progress (all P < 0.05). Measures of segmental hypokyphosis were comparable between groups. As development was tracked at each subsequent scan, coronal and axial plane differences between groups increased in both magnitude and number of differences. Initial disparity and then subsequent increasing magnitude of change of axial rotation may indicate a higher propensity to clinically progress in the future. This knowledge hopes to provide useful management information for AIS care providers and prognostic education for patients alike. II.

The Spectrum of Sarcina Colonization in the Gastrointestinal Tract of Pediatric and Adolescent Patients.

Introduction. Sarcina organisms are rare, gram-positive, sugar-fermenting cocci, identifiable in tissues only by histologic examination or molecular testing. Since its discovery, the pathogenicity and relevance of Sarcina in the human gastrointestinal tract has remained ill-defined. A recent literature review of 66 reported examples demonstrated the potential for severe complications such as emphysematous gastritis and gastric perforation. In pediatrics, colonization is associated with mucosal alterations and/or gastrointestinal dysmotility/obstruction with variable outcomes, including death secondary to gastric perforation. Yet, the features of Sarcina colonization within the gastrointestinal tract of adolescents are poorly understood and rarely reported. Methods. We present the gastrointestinal histopathological findings and the complete history of 4 pediatric patients with Sarcina colonization at our institution. Additionally a literature review with focus in the keywords "Sarcina" and "gastrointestinal' was performed, and the clinical and histopathological features of all previously reported examples of Sarcina in the gastrointestinal tract of pediatric patients were summarized. Results. All 4 patients had delayed gastric emptying, 3 of them due to neurologic disease, and one with pyloric obstruction due to duodenal ulceration with Helicobacter gastritis. In the 3 patients with available esophageal biopsies, it was associated to esophagitis with increased intraepithelial eosinophils. Conclusion. The potential pathogenicity of Sarcina colonization in the gastrointestinal tract of pediatric patients needs to be reevaluated. Due to potential serious complications, the identification of these organisms in the gastrointestinal tract sample should be reported and warrants further evaluation for possible gastrointestinal dysmotility or other mucosal alterations.

Long-term impact of nusinersen on motor and electrophysiological outcomes in adolescent and adult spinal muscular atrophy: insights from a multicenter retrospective study.

5q spinal muscular atrophy (SMA) is a progressive autosomal recessive motor neuron disease. We aimed to assess the effects of nusinersen on motor function and electrophysiological parameters in adolescent and adult patients with 5q SMA. Patients with genetically confirmed 5q SMA were eligible for inclusion, and clinical data were collected at baseline (V1), 63days (V4), 180days (V5), and 300days (V6). The efficacy of nusinersen was monitored by encompassing clinical assessments, including the Revised Upper Limb Module (RULM), Hammersmith Functional Motor Scale Expanded (HFMSE), 6-Minute Walk Test (6MWT), and percent-predicted Forced Vital Capacity in sitting position (FVC%) and Compound Muscle Action Potential (CMAP) amplitude. The patients were divided into "sitter" and "walker" subgroups according to motor function status. 54 patients were screened, divided into "sitter" (N = 22) and "walker" (N = 32), with the mean age at baseline of 27.03years (range 13-53years). The HFMSE in the walker subgroup increased significantly from baseline to V4 (mean change +2.32-point, P = 0.004), V5 (+3.09, P = 0.004) and V6 (+4.21, P = 0.005). The patients in both the sitter and walker subgroup had no significant changes in mean RULM between V1 and the following time points. Significant increases in CMAP amplitudes were observed in both upper and lower limbs after treatment. Also, patients with RULM ≥ 36 points showed significant CMAP improvements. Our analysis predicted that patients with CMAP amplitudes of trapezius ≥ 1.76mV were more likely to achieve significant motor function improvements. Nusinersen effectively improves motor function and electrophysiological data in adolescent and adult patients with SMA. This is the first report on the CMAP amplitude changes in the trapezius after treatment in patients with SMA. The CMAP values effectively compensate for the ceiling effect observed in the RULM, suggesting that CMAP could serve as an additional biomarker for evaluating treatment efficacy.

Anatomic and functional masseter muscle adaptation following orthognathic surgery—MRI analysis in 3 years of follow-up

BackgroundOrthodontic and surgical technical advances in recent years have resulted in treatment opportunities for a whole range of craniofacial skeletal disorders either in the adolescent or adult patient. In the growing child, these can include myofunctional orthodontic appliance therapy or distraction osteogenesis procedures, while in the adult, the mainstay approach revolves around orthognathic surgery.The literature agrees that for a change in craniofacial morphology to remain stable, the muscles acting upon the facial skeleton must be capable of adaptation in their structure and, therefore, their function. Failure of the muscles to adapt to the change in their length or orientation will place undesirable forces on the muscle attachments leading to potential instability of the skeleton. Adaptation can occur through various processes including those within the neuromuscular feedback mechanism, through changes within muscle structure or through altered muscle physiology, and through changes at the muscle/bone interface.It is now accepted that because there is no single method of assessing masticatory function, several measures should be taken, and whenever possible, simultaneously.MethodsThis investigation was designed to apply several, newly developed and more sophisticated methods of measuring muscle structure and function to a situation where adaptation of muscle is pivotal to the success of a therapeutic approach. Patients attending the combined orthodontic/orthognathic surgery clinic at the Clitrofa – Centro Médico, Dentário e Cirúrgico, in Trofa, Portugal, were screened. Ten patients scheduled for a bimaxillary osteotomy involving a combination of maxillary Le Fort I impaction procedure coupled with a sagittal split advancement of the mandible were selected to form the study group.The patients have MRI of the masseter muscle to evaluate the masseter muscle volume and fibre orientation changes. This exam was taken before surgery (T0), 6 to 12 months after surgery (T1), and 3 years after surgery (T2), by two independent observers, according to the protocol jointly developed between the Eastman Dental Institute – University of London and the MRI Centre - Department of Radiology at John Radcliffe Hospital – University of Oxford.ResultsSignificant differences (p < 0.05) have been identified between Time 0 (pre-op) and Time 1 (6–12 months post-op) regarding the masseter area (mm2). The differences against Time 0 (pre-op) seem to disappear at Time 2 (3 years post-op).ConclusionsMRI therefore seems to be a valid tool for measuring differences in the masseter muscle area and volume associated with high-severity occlusal deformities, although showing not to be as efficient in detecting the same differences in cases of low-severity occlusal deformities.

Case Series: Fibula Free Flap with Bone Allograft as the Gold Standard in Lower Limb-Salvage Surgery for Adolescent Patients with Primary Bone Tumors Located within Tibial Diaphysis: Technical Modifications and Short-Term Follow-Up

Case Series: Fibula Free Flap with Bone Allograft as the Gold Standard in Lower Limb-Salvage Surgery for Adolescent Patients with Primary Bone Tumors Located within Tibial Diaphysis: Technical Modifications and Short-Term Follow-Up

Long-term safety and effectiveness of lurasidone in adolescents and young adults with schizophrenia: pooled post hoc analyses of two 12-month extension studies

Background and ObjectivesThe aim of this analysis was to evaluate the long-term safety and effectiveness of lurasidone in the treatment of schizophrenia in adolescents and young adults (13–25).MethodsThe 2 pooled studies used similar designs and outcome measures. Patients (13–25) with schizophrenia completed an initial double-blind 6-week trial of lurasidone (40 and 80 mg/day) in the adolescent trial and (80 and 160 mg/day) in the young adult trial. In open-label long-term trials, adolescent patients were treated with 20–80 mg/day lurasidone, and adults were treated with 40–160 mg/day lurasidone. Efficacy was evaluated based on the Positive and Negative Syndrome Scale (PANSS) and Clinical Global Impression-Severity Scale (CGI-S).ResultsThe safety population consisted of 306 patients (mean age, 16.2 years; 208 patients (68.0%) who completed 12 months of treatment; 8.2% who discontinued treatment by 12 months due to an adverse event). The mean (SD) changes in the PANSS total score from the extension baseline to months 6 and 12 were − 11.8 (13.9) and – 15.3 (15.0), respectively (OC), and the mean (SD) changes in the CGI-S score were − 0.8 (1.0) and − 1.0 (1.1), respectively (OC). The most frequent adverse events were headache (17.6%), anxiety (11.4%), schizophrenia (9.8%), and nausea (9.8%). No clinically meaningful changes were observed in weight, metabolic parameters, or prolactin.ConclusionsIn adolescents and young adults with schizophrenia, treatment with lurasidone was generally well tolerated and effective. Long-term treatment was associated with a continued reduction in symptoms of schizophrenia. Long-term treatment was associated with minimal effects on weight, metabolic parameters, and prolactin.Clinicaltrials.gov identifiers D1050234, D1050302.

Prospective evaluation of nasal septal perforation repair with absorbable plate and temporalis fascia in early adolescent patients

ObjectivesIn the pediatric population, nasal septal perforations (NSP) are rare, and management is not well described. The use of various techniques has been reported in adults, including the use of interposition grafts, however this technique has not been described in the pediatric population.Our hypothesis is that the closure rate using absorbable d-lactide and l-lactide (each 50 %), polymer (PDLLA) plates as interposition grafts with temporalis fascia for NSP repair in the pediatric population will be an effective method compared to previous techniques. MethodsChart review was performed on patients who underwent NSP repair before June 2021, results were compared to a prospective evaluation of an interposition plate graft for repair at a tertiary care children's hospital. ResultsFifteen patients via previous techniques and 5 patients via PDLLA and fascia graft were reviewed. Etiology of perforations included 45 % idiopathic, 25 % traumatic, and 15 % iatrogenic. In the previous techniques group, 10 were male, mean (median) age 14.4 years (15.2). Average size of NSP was 12.6 mm ± 6.6 mm (SD). 14/15 (93 %) patients had resolution of symptoms at 10-week follow-up, and 2/15 (13 %) required repeat repair. Five prospective patients were repaired with a PDLLA and fascia interposition graft, 4 were female, mean (median) age 14.6 years (Nassif and Scott, 2021 Feb 1) [14]. Average size of NSP was 11 mm ± 2.2 mm (SD). 100 % had resolution of symptoms at 10-week follow-up, 0 needed repeat surgery. No significant difference was found in size of NSP or in need for repeat procedure (p > 0.05) between the groups. ConclusionUse of absorbable PDLLA interposition grafts with temporalis fascia for NSP repair in the pediatric population is effective at closing and resolving symptoms associated with NSP.

Bi-valve braces for treatment of pectus carinatum in teenagers: impact on patients quality of life

BackgroundPectus carinatum may be a major issue for adolescent patients, as chest-wall malformations can have a negative impact on body image and induce self-esteem disorders. Bracing has become the first line treatment for adolescent patients whose anterior chest wall is still flexible. The main obstacle is the tolerability of the bracing device along with the patient’s compliance to the device. The aim of this study was to examine the quality of life of adolescent patients treated with bracing to correct pectus carinatum in our institution. From November 2021 to July 2023, 28 patients with pectus carinatum were treated with bracing therapy in our chest wall deformities unit. We used a bivalve brace, manufactured with computer-aided design. All patients were asked to answer a multiple-choice questionnaire designed to assess how bracing therapy impacts their daily life, treatment compliance, and overall satisfaction. Patients who wore the brace for at least 3 months, and at least 5 days per week and 10 h per day, are defined as compliant with the bracing protocol.ResultsThe mean age of the patients was 14 ± 1.36 years. Patients wore the brace for an average of 10.1 ± 6.9 months in the compliance group (n = 18), and 2.2 ± 2.5 months in the non-compliance group (n = 10). Our results show that compliant patients are fairly satisfied of the comfort of the brace, with a limited impact of the device on their daily activities and social life. However, non-compliant patients report the following dissatisfactions regarding the brace: higher pain scores, difficulty in breathing, and issues sleeping with the brace. The compliant patients noted a significant improvement in the appearance of their chest, and were satisfied with the appearance of their chest. The non-compliant patients did not notice any changes.ConclusionsCompliant patients treated with a bi-valve brace for pectus carinatum seem to have a good quality of life with a limited impact on their daily life and social activities. The bi-valve brace also seems to be an effective therapy in compliant patients.

The diagnostic accuracy of community spine radiology for adolescent idiopathic scoliosis brace candidates.

The study aims to establish the diagnostic accuracy of community spine x-rays for brace candidates. A review of adolescent idiopathic scoliosis patients seen for initial visit at a tertiary care pediatric hospital was conducted (n = 170). The index test was the pre-referral community spine x-ray interpreted by a community radiologist. Measures of diagnostic accuracy for the index test were determined against the reference standard if images were obtained within 90 days (n = 111). The reference standard was the 3-foot standing EOS spine x-ray evaluated by spine specialists. Diagnostic criterion for a brace candidate was dichotomized by Cobb angle range (25-40°) according to Scoliosis Research Society criteria. Risser stage was not included given significant missing data in index reports. To mitigate the uncertainty around true progression, sensitivity analyses were conducted on a sub-sample of data when index test was within 60 days of the reference standard (n = 67). Accuracy of the community spine x-ray to detect a brace candidate was 65.8% (95% CI 56.2-74.5). Sensitivity of the index test was 65.4% with a false negative rate of 34.6%. Specificity was 66.1% with a false positive rate of 33.9%. Positive and negative predictive values were 63.0% and 68.4%, respectively. Of the total number of brace candidates (n = 52), 32.7% were missed because of underestimation in Cobb angle (95% CI 21.5-46.2). The proportion of missed brace candidates because of underestimation was unchanged with 60-day data (p = 0.37). Inaccuracies in community spine radiology may lead to missed opportunities for non-operative treatment.

Disparities in the Occurrence of Long-Term Effects of Bone Marrow Suppression after Treatment in Adolescent Young Adult Breast Cancer Survivors.

Many adolescent and young adult (AYA) patients with breast cancer (BC) receive adjuvant therapy as initial treatment, with long-term bone marrow suppression as a potential complication, but no studies have evaluated the impact of race/ethnicity on the development of bone marrow suppression in AYA BC survivors. Female patients ages 15-39 years diagnosed with BC (2006-2018) and surviving ≥ 2 years were identified from the California Cancer Registry and linked to statewide hospitalization data. We estimated the cumulative incidence of developing late effects of bone marrow suppression, such as leukopenia, anemia, thrombocytopenia, bleeding, and infection/sepsis, during hospital discharge diagnoses present ≥ 2 years after diagnosis. We examined the impact of sociodemographic and clinical factors on late effects using multivariate Cox proportional hazards regression. Of 11,293 patients, 42.8% were non-Hispanic (nH) White, 28.8% Hispanic, 19.5% nHAsian/Pacific Islander, and 7.5% nH Black. In multivariable analyses, nH Blacks had the highest risk (versus nH Whites) of anemia [hazard ratio (HR) 1.72, 95% confidence interval (CI) 1.47-2.02], leukopenia (HR 1.56, CI 1.14-2.13), thrombocytopenia (HR 1.46, CI 1.08-1.99), major infection/sepsis (HR 1.64, CI 1.4-1.92), and bleeding (HR 1.89, CI 1.39-2.58). Hispanics had a higher risk of developing anemia (HR 1.17, CI 1.04-1.32), bleeding (HR 1.4, CI 1.12-1.76), and major infections/sepsis (HR 1.36, CI 1.21-1.52). Asian/Pacific Islanders had only a higher risk of developing bleeding (HR 1.33, CI 1.03-1.72). Patients from a low neighborhood socioeconomic status had a 20% higher risk of infection/sepsis (HR 1.21, CI 1.1-1.34), but there were no associations for the other late effects. We identified that AYAs of nH Black, Hispanic, and Asian/Pacific Islander race/ethnicity are at an increased risk of several late effects after adjuvant therapy compared with nH White patients. From these data, providers can implement early/frequent screening of hematologic late effects in these high-risk survivors.

Adult endocrinologists' perspectives on transitioning adolescent patients with congenital adrenal hyperplasia.

The study aimed to evaluate adult endocrinologists' perspectives on caring for patients with congenital adrenal hyperplasia (CAH) and views on their transition from pediatric to adult care. An online survey was conducted among adult clinical endocrinologists at Harvard Medical School-affiliated hospitals from March to October 2022. Most participants (25/34, 73.5 %) treat patients with CAH and expressed moderate to high confidence (23/32, 71.9 %) in their care. Those that did not treat or accept referrals cited insufficient expertise, knowledge, and resources as reasons. Only half of respondents correctly answered at least 50 % ofstandard of care questions. The main transition of care barrieridentified was the absence of standardized policies (12/34, 35.3 %). Participants, though involved in care of patients with CAH, had varied responses to standard of care questions and transition of care barriers, emphasizing the need for standardized transition protocols and additional training to ensure up-to-date clinical knowledge.

Incidence of Ipsilateral Femoral Neck and Shaft Fractures in Pediatric and Adolescent Patients

OBJECTIVES: To identify the incidence, patient characteristics, and effectiveness of radiographic screening methods for detecting ipsilateral femoral neck and shaft fractures in pediatric and adolescent trauma patients. METHODS: Design: Retrospective cohort study. Setting: The study was conducted at a tertiary pediatric trauma hospital. Patient Selection Criteria: Patients under the age of 18 years treated for a femoral shaft fracture between 2004 and 2018 were reviewed. Pathologic (metabolic bone disease or bone lesion), periprosthetic and penetrating traumatic femoral shaft fractures were excluded. Outcome Measurements and Comparisons: Patient demographics, mechanisms of injury, treatment methods, and associated injuries were analyzed. Pre-treatment x-rays and CT scans were reviewed for the identification of ipsilateral femoral neck and shaft fractures. RESULTS: Among 840 pediatric patients included in the study, 4 patients (0.5%) sustained ipsilateral femoral neck and shaft fractures. All of the femoral neck fractures were observed in adolescents (ages 13-17) and involved in high-energy traumas. In adolescents involved in high-energy trauma, the incidence increased to 1.7%. Pre-treatment sensitivity of both x-rays and CT scans was only 50% for the detetion of femoral neck fractures. CONCLUSIONS: This study reveals that ipsilateral femoral neck and shaft fractures in pediatric patients are rare, occurring in adolescents involved in high-energy trauma. The findings suggest the need for a selective, rather than routine, use of CT scans based on the patient's age and the mechanism of injury. The use of alternative imaging methods such as MRI should be considered in order to balance diagnostic accuracy while minimizing radiation exposure. LEVEL OF EVIDENCE: Prognostic- Level III. See Instructions for Authors for a complete description of levels of evidence.

Harmonization of the Upfront Osteosarcoma Treatment Paradigm for Adolescents and Young Adults.

Limited guidance exists on streamlining cancer therapy for adolescent and young adult (AYA) patients 15-39 years of age, as much of the current data are extrapolated from pediatric or adult counterparts and can differ significantly between the two care models. Harmonization of standard treatment approaches has the potential to improve outcomes and establish a foundation for the development of future clinical trials. We present our experience harmonizing treatment and supportive care regimens for AYA patients with osteosarcoma receiving treatment with methotrexate, doxorubicin, and cisplatin (MAP) therapy on the pediatric and adult sarcoma services at the Memorial Sloan Kettering Cancer Center.

Treatment and genetic analysis of multiple supernumerary and impacted teeth in an adolescent patient

BackgroundMultiple supernumerary teeth, combined with numerous impacted teeth, can lead to various malocclusions, posing significant treatment challenges. While certain genes associated with syndromic cases of multiple supernumerary and impacted teeth have been identified, the etiologies of non-syndromic cases still largely remain elusive.Case presentationHere, we report a treatment of a 12-year-old boy who presented with 10 supernumerary teeth and 6 impacted teeth, accompanied by a genetic analysis to explore the underlying etiology. During the treatment, fifteen teeth were extracted, and various skilled techniques, including the closed-eruption technique and the application of by-pass arches, were utilized. Post-treatment, traction was successful for all the impacted teeth, without any tooth mobility or reduction in gingival height. Space closure, well-aligned teeth, and excellent functional occlusion were achieved. Furthermore, comprehensive genetic analysis was conducted through whole-exome sequencing on the patient and his parents, which revealed a potential link between the patient’s numerous supernumerary teeth and abnormal mineralization. Notably, the p.Ser496Pro variant in the TCF7L2 gene was identified as a potential candidate variant in this patient.ConclusionsOverall, our findings not only report the treatment of a rare case involving multiple supernumerary and impacted teeth but also offer valuable insights into the molecular basis of supernumerary teeth.

Priming lymphocyte responsiveness and differential T cell signaling in pediatric IBD patients with Cannabis use.

This observational study examines the impact of chronic cannabis use on peripheral immune cell function in adolescent IBD patients from Children's Hospital Colorado. Cannabis users displayed altered T cell phenotype, increased pro-inflammatory cytokine release and dephosphorylation of protective protein kinases.

Guardian Reasons for Accessing Their Transgender and Gender-Diverse Adolescent's Patient Portal Account

PurposeTo understand if and why guardians access their adolescent child's electronic health record patient portal account. MethodsGuardians of transgender and gender-diverse adolescents completed a survey regarding patient portal use. Descriptive statistics were used to describe items related to guardian access to adolescent portal accounts. ResultsOf 82 respondents, 37.8% indicated they had used their child's login to access the patient portal. Most indicated they accessed their adolescent's account because their child asked them to do so. Other common reasons included being worried they might miss important health information and not realizing there was a difference between patient and proxy accounts. DiscussionResults of this study provide a more detailed understanding regarding guardian access to adolescent patient portals. Findings can be used to inform adolescent patient portal design and enrollment practices that protect adolescent confidentiality.

Case report: Challenging kidney transplantation in an adolescent patient with tetralogy of Fallot

Case report: Challenging kidney transplantation in an adolescent patient with tetralogy of Fallot

Essential components of an effective transition from paediatric to adult neurologist care for adolescents with Duchenne muscular dystrophy; a consensus derived using the Delphi methodology in Eastern Europe, Greece and Israel

PurposeAn increasing number of patients with Duchenne muscular dystrophy (DMD) now have access to improved standard of care and disease modifying treatments, which improve the clinical course of DMD and extend life expectancy beyond 30 years of age. A key issue for adolescent DMD patients is the transition from paediatric- to adult-oriented healthcare. Adolescents and adults with DMD have unique but highly complex healthcare needs associated with long-term steroid use, orthopaedic, respiratory, cardiac, psychological, and gastrointestinal problems meaning that a comprehensive transition process is required. A sub-optimal transition into adult care can have disruptive and deleterious consequences for a patient’s long-term care. This paper details the results of a consensus amongst clinicians on transitioning adolescent DMD patients from paediatric to adult neurologists that can act as a guide to best practice to ensure patients have continuous comprehensive care at every stage of their journey.MethodsThe consensus was derived using the Delphi methodology. Fifty-three statements were developed by a Steering Group (the authors of this paper) covering seven topics: Define the goals of transition, Preparing the patient, carers/parents and the adult centre, The transition process at the paediatric centre, The multidisciplinary transition summary – Principles, The multidisciplinary transition summary – Content, First visit in the adult centre, Evaluation of transition. The statements were shared with paediatric and adult neurologists across Central Eastern Europe (CEE) as a survey requesting their level of agreement with each statement.ResultsData from 60 responders (54 full responses and six partial responses) were included in the data set analysis. A consensus was agreed across 100% of the statements.ConclusionsIt is hoped that the findings of this survey which sets out agreed best practice statements, and the transfer template documents developed, will be widely used and so facilitate an effective transition from paediatric to adult care for adolescents with DMD.

Essential components of an effective transition from paediatric to adult neurologist care for adolescents with Duchenne muscular dystrophy; a consensus derived using the Delphi methodology in Eastern Europe, Greece and Israel

PurposeAn increasing number of patients with Duchenne muscular dystrophy (DMD) now have access to improved standard of care and disease modifying treatments, which improve the clinical course of DMD and extend life expectancy beyond 30 years of age. A key issue for adolescent DMD patients is the transition from paediatric- to adult-oriented healthcare. Adolescents and adults with DMD have unique but highly complex healthcare needs associated with long-term steroid use, orthopaedic, respiratory, cardiac, psychological, and gastrointestinal problems meaning that a comprehensive transition process is required. A sub-optimal transition into adult care can have disruptive and deleterious consequences for a patient’s long-term care. This paper details the results of a consensus amongst clinicians on transitioning adolescent DMD patients from paediatric to adult neurologists that can act as a guide to best practice to ensure patients have continuous comprehensive care at every stage of their journey.MethodsThe consensus was derived using the Delphi methodology. Fifty-three statements were developed by a Steering Group (the authors of this paper) covering seven topics: Define the goals of transition, Preparing the patient, carers/parents and the adult centre, The transition process at the paediatric centre, The multidisciplinary transition summary – Principles, The multidisciplinary transition summary – Content, First visit in the adult centre, Evaluation of transition. The statements were shared with paediatric and adult neurologists across Central Eastern Europe (CEE) as a survey requesting their level of agreement with each statement.ResultsData from 60 responders (54 full responses and six partial responses) were included in the data set analysis. A consensus was agreed across 100% of the statements.ConclusionsIt is hoped that the findings of this survey which sets out agreed best practice statements, and the transfer template documents developed, will be widely used and so facilitate an effective transition from paediatric to adult care for adolescents with DMD.

Assessing Psychological Disorders in Turkish Adolescents with Transfusion-Dependent Thalassemia

We investigated depression and anxiety levels and related psychological disorders in adolescents with transfusion-dependent thalassemia (TDT) in this study. The study was conducted in two pediatric hematology outpatient clinics and included adolescents with TDT (14.8 ± 2.4 years, n = 40) in the study and compared them with the healthy age-matched control group (14.3 ± 2.3 years, n = 62). The Turkish version of the Revised Child Anxiety and Depression Scale (RCADS) was used to determine depression, anxiety, and related psychologic disorders (obsession, panic disorder, social phobia). Depression, anxiety, obsession, panic disorder, and social phobia scores were significantly higher in the patient group compared with the control (all p &lt; 0.05). Ferritin levels were positively correlated with total depression, general anxiety, separation anxiety, and social phobia scores, but transfusion frequency and young age were the confounding factors. Patients in early adolescence and those who require more frequent blood transfusions are at higher risk of developing psychological disorders; routine screening for mood disorders should be warranted. Serum ferritin level may be a good warning indicator for early recognition of psychologic disorders in TDT patients.