REMS pharmacy tasks: The adoption of an innovative electronic support system.

OBJECTIVES: The objective of this study was to evaluate the pharmacy adoption rates of an online Risk Evaluation and Mitigation Strategy (REMS) Pharmacy Portal designed as an alternative for REMS-certified pharmacies to perform mandatory pharmacy dispense confirmations and to assess whether Pharmacy Portal uptake was affected by the pharmacy daily dispense volume. SETTING: REMS-certified pharmacies dispensing lenalidomide (Revlimid), pomalidomide (Pomalyst), or thalidomide (Thalomid). PRACTICE DESCRIPTION: Primarily specialty and hospital pharmacies in the limited distribution network that used the REMS Pharmacy Portal. PRACTICE INNOVATION: A self-service pharmacy portal was developed to allow REMS-certified pharmacies to obtain confirmation numbers instead of calling a Celgene Customer Care Representative (CCR) or using the Interactive Voice Response System (IVR) system. EVALUATION: The numbers of pharmacy dispense confirmations obtained were identified, and the mean percentages by quarter (Q) were calculated from January 2013 through August 2016 for the Pharmacy Portal, Celgene CCR, and IVR. RESULTS: In Q1 2013, the CCR and IVR options were used for 57% and 43% of dispense confirmations, respectively. After the training period, the Pharmacy Portal rapidly became the most used option (67% of confirmations from Q2 2014). By August 2016, data displayed the continued preference for the pharmacy portal (98% of all confirmations) regardless of the daily dispense volume of the pharmacy. As of March 30, 2018, the pharmacy portal continued to be the preferred option for all pharmacies (maintained at 98.5% use). CONCLUSION: There is an overwhelming REMS pharmacy preference to use the pharmacy portal over the IVR and CCR options, irrespective of the pharmacy daily dispense volume. The rapid uptake of the pharmacy portal is most likely attributed to robust and comprehensive 1-on-1 training and support provided by the REMS sponsor to the REMS-certified pharmacies, but also because it might be easier to use than the other options, resulting in reduced REMS burden for the pharmacy.

Parent-reported health-related quality of life of children with Down syndrome: a descriptive study.

AIM: To describe health-related quality of life of Australian children and adolescents with Down syndrome and compare it with norm-referenced data. METHOD: A cross-sectional survey was conducted with parents of 75 children and adolescents (43 males, 32 females) with Down syndrome aged 5 to 18 years (mean age 13y 2mo, SD 4y 8mo). The proxy-report KIDSCREEN-27 questionnaire was administered and five dimensions of health-related quality of life were measured. Data were analysed descriptively and compared with normative data. RESULTS: Total group mean scores for psychological well-being, autonomy and parent relation, and school environment dimensions were within normal threshold values, whereas mean scores for physical well-being, and social support and peers dimensions, were poorer. For participants with Down syndrome aged 8 to 18 years, the difference with normative data for proxy-reported physical well-being, psychological well-being, and social support and peers dimensions favoured typically developing children. Adolescents (13-18y) with Down syndrome scored poorer on all dimensions than children (5-12y) with Down syndrome. INTERPRETATION: Our findings assist a better understanding of the lived experiences of children and adolescents with Down syndrome, as perceived by their parents, and suggest aspects of health that could be influenced to optimize their quality of life. WHAT THIS PAPER ADDS: Proxy-reported psychological well-being and autonomy were within the normal range for children with Down syndrome. Physical well-being and social support scores were significantly lower than normative data. Proxy-reported scores for adolescents with Down syndrome were consistently poorer than for children with Down syndrome and the differences were clinically important.

Safety of bloodless autologous stem cell transplantation in Jehovah's Witness patients.

Due to the curative potential and improvement in progression-free survival (PFS), high-dose chemotherapy followed by autologous stem cell transplantation (ASCT) is considered the standard of care for several hematologic malignancies, such as multiple myeloma, and lymphomas. ASCT typically involves support with blood product transfusion. Thus, difficulties arise when Jehovah's Witness patients refuse blood transfusions. In order to demonstrate the safety of performing "bloodless" ASCT (BL-ASCT), we performed a retrospective analysis of 66 Jehovah's Witnesses patients who underwent BL-ASCT and 1114 non-Jehovah's Witness patients who underwent transfusion-supported ASCT (TF-ASCT) at Cedars-Sinai Medical Center between January 2000 and September 2018. Survival was compared between the two groups. Transplant-related complications, mortality, engraftment time, length of hospital stay, and number of ICU transfers were characterized for the BL-ASCT group. One year survival was found to be 87.9% for both groups (P = 0.92). In the BL-ASCT group, there was one death prior to the 30 days post transplant due to CNS hemorrhage, and one death prior to 100 days due to sepsis. Based on our data, BL-ASCT can be safely performed with appropriate supportive measures, and we encourage community oncologists to promptly refer JW patients for transplant evaluation when ASCT is indicated.

Co-occurrence of birth defects and intellectual disability.

This study used population-based databases to ascertain birth defects and intellectual disability (ID), defined as full IQ < 70, in children born in Western Australia during 1980-99. Of the children surviving to 1 year (n = 474 285), 4.9% had birth defects and 1.3% ID. ID was identified in 7.9% of children with birth defects. After adjusting for sex, mother's age, race, parity, plurality, birthweight and gestational age the prevalence ratio (PR) [95% confidence interval (CI)] for ID in children with birth defects compared with those with no birth defects was 7.6 [7.2, 8.0]. Those with chromosomal anomalies comprised 3.2% of the group with birth defects. The percentage ID (and PR [95% CI]) in specific categories were: Down's syndrome 97% (84.5 [79.4, 90.0]), sex chromosome anomalies 30.3% (31.0 [23.8, 40.3]), other chromosomal anomalies 64.2% (54.2 [47.2, 62.3]). Birth defects were categorised according to system in the 96.8% of children with non-chromosomal anomalies. The percentage with ID (and PR [95% CI]) for birth defects in each system were: spina bifida 18.8 (16.7 [12.2, 23.0]); nervous (except spina bifida) 38.6 (33.4 [30.3, 36.9]); cardiovascular 4.2 (4.1 [3.5, 4.8]); gastro-intestinal 2.2 (2.0 [1.5, 2.7]); urogenital 2.6 (2.4 [2.0, 2.8]; musculo-skeletal 3.6 (4.0 [3.5, 4.6]); other non-chromosomal 7.0 (7.3 [6.5, 8.3]); and multiple systems 12.3 (10.2 [8.6, 12.2]). Birth defects were present in 30.2% of children with ID (27.7% of children with mild/moderate ID (IQ 40-69) and 54% of children with severe ID (IQ < 40)). Adjusted PRs for birth defects in children with any ID, mild/moderate ID and severe ID compared with children with normal intellectual function were 6.0 [5.8, 6.3], 5.5 [5.3, 5.8] and 10.5 [9.7, 11.4] respectively. The data are useful for those providing services for children with developmental disabilities especially for predicting family support and respite and accommodation requirements for children and adults with severe ID.