Single-centre prospective clinical and procedural analysis identifying causes of delay to haematopoietic stem cell transplant in MPS I (Hurler syndrome) patients

The international standard treatment for mucopolysaccharidosis type I - Hurler syndrome (MPS1H) is haemopoietic stem cell transplant (HSCT) preceded by intravenous enzyme replacement therapy (ERT), with HSCT ideally undertaken before 18 months age to achieve best outcome. The invasive nature and high risk of morbidity and mortality associated with HSCT, in addition to a complex patient cohort, demands an extensive pre-transplant work-up to minimise risks where avoidable. This is achieved by collaboration between transplant and specialist paediatric LD-metabolic services. Transplant may be delayed due to clinical complications pre-transplant, but non-clinical disruptions have also been encountered in practice causing delays from time of diagnosis to transplantation. This work aimed to identify clinical complications and non-clinical disruptions in this process, and to identify areas of improvement for clinical practice, ultimately to achieve timely intervention and optimise clinical outcomes. A single-centre prospective clinical and procedural analysis of 7 MPS1H patients undergoing HSCT between April 2020 - January 2021 was completed. Age at diagnosis (median(range)) was 10 (1.5–25) months. Time from diagnosis to starting ERT (median(range)) was 10 (3–26) days. Time from diagnosis to transplant (median(range)) was 158 (101–189) days, with age at transplant 14 (6.5–30) months. Multiple reasons causing delay were identified. Clinical factors included presence of dilated cardiomyopathy, requirement for adenotonsillectomy to treat obstructive sleep apnoea, Covid-19 infection, vascular device infection, and acute neurosurgical issues including hydrocephalus requiring ventriculoperitoneal shunt and cervical spine stenosis requiring decompression. Non-clinical factors identified included late cancellation of required investigations, missed clinic appointments, and issues with accessing HSCT donors due to UK/European political situation and Covid-19 restrictions. Clear communication between teams was found to be a key identifying factor in ensuring timely completion of the pre-HSCT.

Patient Perceptions of Drive Through Medical Treatment Facilities During the COVID-19 Pandemic

Study Objectives: The cumulative burden of the COVID-19 virus on the US healthcare system is substantial. To help mitigate this burden, novel solutions including tele-health and dedicated screening facilities have been utilized. However, there is limited data on the efficacy of such models and none assessing patient comfort levels with these changes in health care delivery. The aim of our study was to evaluate patients’ perceptions of a drive through medical treatment system in the setting of the COVID-19 pandemic. Methods: In response to the COVID-19 pandemic, NMCP’s emergency department established a drive-through medical evaluation facility (DMEF) in proximity to the emergency department. The DMEF was designed to allow full evaluation of patients to include: vital signs, complete medical history, clinician physical examination, limited point-of-care testing and medication distribution. All patients presenting to the emergency department with symptoms of potential COVID etiology and deemed non-critical were directed to the DMEF for initial evaluation. Patients were surveyed about their experience following their visit. An anonymous questionnaire consisting of 5 questions, utilizing a 5-point Likert scale was distributed via electronic tablet. Results: A total of 827 responses were obtained over two months. Three quarters of respondents believed care received was similar to that in a traditional emergency department. Overall positive impression of the drive-thru was 86.6%, and 95% believed that it was more convenient. Conclusion: Overall the drive-thru medical system was perceived as more convenient than the emergency department, and is viewed as a positive experience. While a dramatic change in the delivery model of medical care, if such systems are able to provide comparable levels of care, they may represent a viable option for sustained and surge health care delivery.

Delivering bronchiectasis physiotherapy clinics remotely: Patient perceptions and future preferences

Background/Aims: National guidelines recommend that patients with bronchiectasis should be reviewed by specialist physiotherapists (Polverino et al., 2017). These appointments should involve teaching of individualised airway clearance techniques, promotion of exercise and education to optimise self-management. During the covid pandemic, face to face appointments were cancelled. Ways of conducting effective physiotherapy consultations remotely were required. We used telephone and video respiratory physiotherapy consultations. We assessed patient satisfaction with remote consultations and views on future modes of clinic delivery. Methods: All patients contacted for a predetermined physiotherapy clinic between 21/4/20 and 29/6/20 were asked questions regarding their consultation and preferences (Table 1). Telephone and video calls were carried out by PM. Data was collected via follow up phone calls using a pre-selected questionnaire or postal questionnaire. Data was recorded and analysed using Excel plus thematic analysis for free text responses. Results: Thirty telephone and 35 virtual consultations were offered. 12 virtual consultations were converted to telephone due to lack of internet access. Thirty-nine (60%) were new referrals, 26 (40%) were reviews. Median age was 65 (range 21-91). Median telephone call duration was 29 minutes (range 15-40). Beyond covid-19 restrictions, twenty-four (37%) preferred a virtual appointment;twenty-two (34%) telephone, four (6%) face to face consultation and fifteen (23%) had no preference. Conclusions: The majority of respondents (97%) were satisfied with remote consultation during covid restrictions. Interestingly, only 6% preferred to return to face to face (Table presented) appointments. Qualitative exploration of reasoning behind these decisions revealed two main themes: convenience and practicality and medical reasons and beliefs. In our cohort, 71% of patients preferred to continue remote physiotherapy clinics. Individual preferences on format and location of care are key components of NHS plans. Our data show that patient satisfaction can be maintained with remote delivery of respiratory physiotherapy, yet more work is needed to standardise and improve remote physiotherapy interventions. In addition to patient preference, ascertaining the comparative clinical effectiveness of appointment formats in the longer term will facilitate evidence-based provision of physiotherapy resources.