ABSTRACT
Background : rIX-FP, a recombinant factor IX (rFIX) linked with recombinant human albumin, has demonstrated efficacy in prophylactic regimens up to every 21 days in patients with hemophilia B. Aims : To describe real world experience for a rural pediatric patient during the COVID-19 pandemic. Methods : Demographic, clinical (HEAD-US and Hemophilia Joint Health Score) and pharmacokinetic (PK) data were collected for up to one year following a switch from rFIX to rIX-FP. Results : In May 2018, a 3-year-old patient (moderate hemophilia B, FIX: 2.7 IU/dL) experienced hemarthrosis in the left knee and initiated prophylaxis with rFIX (40 IU/kg 2×/week). In March 2020, the patient switched to weekly rIX-FP prophylaxis (40 IU/kg), due to difficulties with venous access and accessing the clinic during the pandemic. Trough levels of 7.7% two weeks post-infusion and 5.1% 20 days post-infusion were maintained. Functional and joint assessment on day 90 of rIX-FP treatment showed no change, and no FIX inhibitors developed. On day 95 of treatment, a post-traumatic otorrhagia subsided after a single 40 IU/kg dose of rIX-FP. On day 120, the treatment interval was extended to 10 days without complications. After 160 days, the patient was diagnosed with an asymptomatic SARS-CoV-2 infection and self-isolated at home for 14 days with no changes to prophylactic treatment. On day 200, after a new PK study (trough levels of 6.9% at 14 days post-infusion), the prophylaxis interval was prolonged to 70 IU/kg every 14 days without incident. With 14-day dose intervals, monthly consumption reduced by 56.2% and the number of infusions by 75% compared with the initial rFIX treatment, without affecting efficacy or adherence. Conclusions : Switching to rIX-FP in this pediatric patient reduced the number of administrations and clinic visits without affecting efficacy or adherence, a fact that is relevant in the current context of the pandemic.
ABSTRACT
Background : Haemophilia A (HA) is a disorder characterized by deficiency in clotting FVIII that affects daily lives of patients and caregivers. Emicizumab is a humanized, bispecific, monoclonal antibody, which substitutes missing activated FVIII. Aims : Effectiveness and safety on emicizumab in clinical practice. Methods : Hemolife study initiated in March 2020, describes the socio-occupational impact of HA without inhibitors in patients and caregivers, in terms of quality of life, activity, and productivity/absenteeism, among others. At that time, both COVID-19 pandemic and emicizumab approval for PwHA without inhibitors happened. A multidisciplinary Scientific Committee of experts in HA, rethought Hemolife study to include a cohort of patients treated with emicizumab (Hemolution), resulting in a more complete and stronger RWE study, and avoiding logistical duplications that a separate study would mean to centers and patients. 2020 has been a year for adaptation, efficiency and re-design;also, in RWE context. 100 PwHA will be followed for 12 months within Hemolife. At any time, a patient prescribed with emicizumab may be enrolled in Hemolution cohort, followed for at least 8 months after initiation of emicizumab to collect efficiency and safety data (Figure 1). Finally, Hemolife involves telemedicine;in a pandemic setting, Hemolution could also benefit from this live data collection and may shape a new and more dynamic form of care in HA that avoids being in the hospital. Results : Ongoing study. Protocol was submitted and approved by authorities/ethics committee's (Table 1). Conclusions : By this novel approach we will gather information on emicizumab in clinical practice and provide relevant information to the Haemophilia community, contributing to improve patient care in HA.
ABSTRACT
Introduction: The pandemic has affected various levels of health assistance and we have to wait to know all the aspects that COVID-19 has entailed. Surgeries, rehabilitation, teaching programs, inclusion in clinical trials, pharmacokinetics (PKs), and expanded coagulation studies were suspended. Since next working day after lockdown we organized assistance through teleconferences with the staff working onsite, Clinical Research Associates (CRA) and sponsors, Patient's Association and with people with hemophilia and their caregivers. Also, we had to deal with the stop in 3 trials affecting 5 patients. Our Clinical Trials Unit (CTU) has 2 study nurses, 1 study coordinator, 4 hematologist investigators and 4 researchers, having 59 clinical trials and observational studies open, with more than 200 patients. Our aim is to identify the non-priority procedures affected Methods: We evaluated the assistance provided by the CTU of Hospital Universitario La Paz, from February to September. Assistance was categorized as priority procedures (administration of treatment, urgencies, telephonic follow-up visits and home deliveries of treatment) and non-priority procedures (PKs, onsite follow-up visits, screening/baselines, monitoring visits by CRAs and health education using apps/devices) Results: Since March 16th only 3 of 11 members of the CTU were onsite, 2 of them attending patients with COVID-19 outside our Unit. We performed 17 priority procedures and 0 non-priority. In April, we started going one day/week alternating between staff members. We performed 9 priority procedures and 0 non-priority procedures. In May, we started going twice/week, alternating staff members and performing 26 priority procedures (13 with home delivered treatment) and 4 non-priority procedures. In June, all the staff began to work onsite performing 14 priority procedures and 10 non-priority procedures. During holiday with less staff onsite, we performed 5 priority procedures and 29 non-priority procedures. In September, all staff were working onsite and we performed 3 priority procedures and 38 non-priority Discussion/Conclusion: Only in 2 of the 6 months of observation the whole staff from the CTU was onsite. This stopped and delayed non-priority procedures very important for diagnosis and management. We spent 3 months without performing a single PK, screening or baseline. We also have to consider that delays in clinical trials will affect the availability of products on the market.
ABSTRACT
In times of crisis, continuous adaptation is necessary. Communication between all members of a research team is key to adapting the development of clinical trials to the context of the epidemiological crisis of coronavirus. We are accustomed to performing day-to-day tasks to fulfil protocol requirements with precision. To this end, we typically have had protocol-guided face-to-face visits and we have followed a detailed sequenced procedure during each visit.