Objectives: Demonstration of age appropriate disease and treatment knowledge is important in preparing adolescents with hemophilia for transition to adult care. Our pediatric hemophilia treatment ...center (HTC) aimed to use quality improvement (QI) methodology to develop a tool to assess and document transition skills in order to foster medical independence in a complex healthcare environment.
Methods: A standardized transition tool questionnaire was developed for adolescent/young adult patients diagnosed with hemophilia A or B followed in the Nationwide Children's Hospital HTC. Key drivers for the project included; identifying age appropriate tool elements, development of an educational resource repository, development of communication strategies and development of educational strategies to address knowledge gaps identified through tool use. The Ohio State University adult HTC actively collaborated in tool development. A final 19 item paper tool was devised to assess the knowledge and skills of adolescent patients in the domains of fundamental hemophilia knowledge (6 items) and hemophilia treatment knowledge and skills (13 items). Each item had a potential score of 0 to 2 for a total score range of 0 to 36 points. An item score of 0 indicated an incorrect, incomplete or blank answer, 1 meant the patient could answer with assistance of family or staff and 2 represented an independently derived correct answer. Patients with hemophilia A or B aged 15-21 years who were seen for comprehensive hemophilia care were eligible to complete the tool with each comprehensive visit up to twice per year. Patients with mild and moderate disease are seen once per year while those with severe disease are seen twice per year. The tool was administered by hemophilia staff using a standard script. Once the tool was completed by the patient the hemophilia staff reviewed the answers with the patient and family and provided directed education for incorrectly answered items. The tool was scored independently by a hemophilia nurse and physician. If there was a discrepancy in scoring the answer was reviewed and the score was adjudicated. The final score results were entered into a password protected data collection instrument. Scores between patient groups were compared using a Mann-Whitney test.
Results: The tool was introduced into clinic April 5, 2016 and data was collected through June 5, 2017. During the review period there were 28 eligible patients seen for comprehensive care and 27 patients completed the tool. In 1 case the source document of a completed tool was lost so this patient was eliminated from analysis. Of the remaining 26 patients there were a total of 36 tools to evaluate, 18 patients completed the tool during 1 clinic visit, 6 completed the tool at 2 separate successive visits and 2 patients completed the tool at 3 successive clinic visits. Twenty-five patients were male, and 1 was female, 17 had hemophilia A and 9 had hemophilia B, 15 had severe hemophilia, 7 had moderate disease and 4 had mild disease. The median baseline score for patients with mild and moderate disease (n=15) was 14 points which was significantly lower (p=0.0156) than those with severe hemophilia (n=21) who had a median score of 27 points. Of the 8 patients with multiple assessments, 5 improved their scores by an average of 9 points, and 3 patients had decreases in scores by an average of 5 points.
Conclusions: Quality improvement methodology was used to successfully develop, implement and sustain use of a standardized transition tool in a pediatric HTC. The project identified knowledge and skill gaps and targeted educational opportunities in addition to fostering improved communication between staff at the pediatric and adult HTCs regarding the important topic of transition of care. The scores highlighted that patients with milder disease were at risk for less adequate knowledge about their hemophilia care than those with severe disease. Next steps include continued utilization of the tool and ongoing evaluation of scores to determine if the tool fosters sustained improvement in disease and treatment understanding as well as development of tools to span the age continuum. Ongoing collaboration between the adult and pediatric HTCs will be needed to determine if the tool increases successful transition to adult care.
Dunn:Shire: Consultancy, Other: Unrestricted educational grant, Research Funding; Biogen: Other: Unrestricted educational grant, Research Funding; Kedrion: Other: Unrestricted educational grant; CSL Behring: Consultancy, Other: Unrestricted educational grant; Bayer: Consultancy, Other: Unrestricted educational grant; World Federation of Hemophilia USA: Membership on an entity's Board of Directors or advisory committees; NovoNordisk: Other: Unrestricted educational grant; Octapharma: Other: Unrestricted educational grant; Alnylam: Other: Unrestricted educational grant. Casto:Shire: Speakers Bureau. Biega:Shire: Speakers Bureau.
Persons with mild hemophilia A (HA) may use intranasal desmopressin prior to sports participation. Desmopressin is expensive and can cause vomiting, headache, palpitation, and occasionally seizures. ...Our group has previously documented a 2.3-fold increase in factor VIII activity (FVIII:C) in adolescents with mild HA after moderate-intensity aerobic exercise. Herein, we report principal findings of a randomized trial of intranasal desmopressin vs a standardized, moderate-intensity aerobic exercise regimen in adolescents with mild HA. Our primary objective was to compare the change in FVIII:C associated with these 2 interventions. We also examined changes in hemostatic parameters arising from their sequential administration. The study was conducted simultaneously at the Hospital for Sick Children, Canada, and Nationwide Children's Hospital, USA. Thirty-two eligible male adolescents (mean age ± standard deviation: 16.1 ± 2.6 years) with mild HA (mean baseline FVIII:C: 27.9% ± 18.4%) were randomized to 1 of 4 study arms (desmopressin followed by exercise, desmopressin alone, exercise followed by desmopressin, and exercise alone). Blood work was obtained at baseline and at 3 subsequent time-points. Participants randomized to exercise cycled on an ergometer for approximately 12 minutes, with the final 3 minutes at 85% of their predicted maximum heart rate. Standard weight-based dosing of desmopressin was used. Mean immediate increase in FVIII:C was 1.7-fold with exercise compared with 1.9-fold with desmopressin (noninferiority, P = .04). Exercise-induced improvement in hemostatic parameters including FVIII:C was brief compared with more sustained improvements seen with desmopressin. More than 60% of participants randomized to receive both exercise and desmopressin achieved normal (>50%) FVIII:C, 75 and 135 minutes into the study protocol.
Introduction
Multidisciplinary clinics in academic settings are often inefficient and can lead to lengthy clinic visits for patients and staff.
Aim
We aimed to use quality improvement (QI) ...methodology and a multidisciplinary approach to optimize outpatient comprehensive haemophilia clinic flow.
Methods
At baseline, a multidisciplinary QI team created a key driver diagram to identify drivers of haemophilia clinic flow. Identified drivers included patient needs/scheduling, provider flow and laboratory/research requirements. From December 2016 to August 2017, value stream mapping (VSM) was used to identify barriers to clinic flow, and plan–do–study–act cycles were used to address these barriers. Interventions included (a) standardizing the order in which providers saw patients to enable time‐sensitive laboratories, (b) improving HTC team meeting functionality, (c) optimizing a visual management board and implementing a flow coordinator, (d) initiating a team huddle prior to clinic start and (e) modifying the clinic appointment template. Timely laboratory draw was used as a surrogate marker of clinic flow, and VSM utilization percentage was used as an objective measure of efficiency.
Results
We did not demonstrate a statistically significant improvement in timed laboratory draws; however, clinic utilization percentage increased by 30%, which resulted in adding point‐of‐care musculoskeletal ultrasound services without lengthening clinic duration.
Conclusion
Quality improvement methodology is an effective means of improving clinic utilization in a multidisciplinary clinic.
► VDR interacting proteins (VIPs) bind to VDR to yield genomic and nongenomic effects. ► We identified new VIPs via a yeast two-hybrid screen of a human heart library. ► These novel VIPs include ...CXXC5, FASTK, NR4A1, TPM2, MYL3 and XIRP1. ► We demonstrated the activity and interaction of the VIPs with VDR in vivo. ► CXXC5 is a potent and dose-dependent modulator of VDR/VDRE-mediated transcription.
The nuclear vitamin D receptor (VDR) modulates gene transcription in 1,25-dihydroxyvitamin D3 (1,25D) target tissues such as kidney, intestine, and bone. VDR is also expressed in heart, and 1,25D deficiency may play a role in the acceleration of cardiovascular disease. Employing a yeast two-hybrid system and a human heart library, using both a 1,25D-independent and 1,25D-dependent screen, we discovered six candidate VDR interacting proteins (VIPs). These novel VIPs include CXXC5, FASTK, NR4A1, TPM2, MYL3 and XIRP1. Mammalian two-hybrid assays as well as GST pull-downs were used to confirm VIP–VDR interaction, and the combination of these two assays reveals that CXXC5, XIRP1, FASTK and NR4A1 interactions with VDR may be modulated by 1,25D. The functional effects of these VIPs on 1,25D-mediated gene expression were explored in transcriptional assays employing three separate and distinct 1,25D-responsive element (VDRE)-driven luciferase reporter genes in transfected Caco-2 and HEK-293 cells, and in a C2C12 myoblast line. FASTK and TPM2 activated expression in all cell line and promoter contexts, while CXXC5 and XIRP1 exhibited differing effects depending on the cell line and promoter employed, suggesting promoter and cell-specific effects of these unique VIPs on VDR signaling. Further evaluation of the interaction between CXXC5 and VDR revealed that CXXC5 acts in a dose-dependent manner to stimulate VDR-mediated transcription on select VDREs. Identification of novel heart VIPs and their influence on VDR activity may increase our understanding of how vitamin D impacts cardiac physiology and may facilitate development of VDR/VIP drug analogs to combat heart disease.
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