Acute complications in children with sickle cell disease: Prevention and management.

Sickle cell disease (SCD) is a chronic, multi-system disease that requires comprehensive care. The sickling of red blood cells leads to hemolysis and vascular occlusion. Complications include hemolytic anemia, pain syndromes, and organ damage. Patterns of immigration and an increase in newborn screening mean that paediatric health care providers across Canada, in small and large centres alike, need to be knowledgeable about SCD. This statement focuses on principles of prevention, advocacy, and the rapid treatment of common acute complications. Guidance includes the current status of newborn screening, recommendations for immunizations and antibiotic prophylaxis, and an introduction to hydroxyurea, a medication that reduces both morbidity and mortality in children with SCD. Case vignettes demonstrate principles of care for common acute complications of SCD: vaso-occlusive episodes (VOE), acute chest syndrome (ACS), fever, splenic sequestration, aplastic crises, and stroke. Finally, principles of blood transfusion are highlighted, along with indications for both straight and exchange blood transfusions.

Comparison of two vitamin D supplementation strategies in children with sickle cell disease: a randomized controlled trial.

Previously, we showed that nearly 70% of children followed in our sickle cell disease (SCD) clinic were vitamin D- deficient and had low vitamin intake with poor use of supplements. We compared the change in serum 25-hydroxyvitamin D [25(OH)D], safety and clinical impact of two vitamin D supplementation regimens in children with SCD. Children (5-17 years, all genotypes) were randomized to a single bolus of vitamin D3 (300 000 IU; n = 18) or placebo (n = 20). All children received a prescription for daily 1 000 IU vitamin D3 . Serum 25(OH)D and calcium, urinary calcium/creatinine ratio, musculoskeletal pain, quality of life, haematology and bone markers were assessed at baseline and three months post intervention. Bolus administration led to a greater rise in 25(OH)D levels from baseline compared to placebo (20 ± 16 nmol/l vs. 2 ± 19 nmol/l; P = 0·003) and correction of vitamin D deficiency. No hypercalcaemia nor hypercalciuria occurred during the study, but more children in the bolus group experienced gastrointestinal symptoms within the first month (P = 0·04). There were no differences between groups for other outcomes. The use of a high-dose vitamin D bolus combined with daily 1 000 IU vitamin D3 was more efficient in raising 25(OH)D levels than daily supplementation alone in children with SCD.

Changes in hair cortisol and self-reported stress measures following mindfulness-based stress reduction (MBSR): A proof-of-concept study in pediatric hematology-oncology professionals.

BACKGROUND AND PURPOSE: Little data is available on the effect of mindfulness amongst pediatric hematology-oncology professionals. The purpose was to further document change in biological and psychological stress following a mindfulness-based stress reduction (MBSR) program. MATERIALS AND METHODS: We led two pre-post interventional studies (n = 12 and n = 25) and measured changes on hair cortisol concentrations, perceived stress, psychological distress and burnout. RESULTS: Professionals did not change on biological stress (d = 0.04), but improved on self-reported measures (median d = 0.58). Effects were maintained over 3 months for psychological distress, anxiety, depression, and burnout (median d = 0.66). Effects were larger if trainees participated to the retreat and if they reported higher baseline perceived stress. CONCLUSION: In pediatric hematology-oncology professionals, an MBSR program was related with improvements in self-reported stress over 3 months. Components of the program and characteristics of trainees may influence the impact of MBSR.

Clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History Study.

An international, multicenter registry was established to collect retrospective and prospective clinical data on patients with pyruvate kinase (PK) deficiency, the most common glycolytic defect causing congenital nonspherocytic hemolytic anemia. Medical history and laboratory and radiologic data were retrospectively collected at enrollment for 254 patients with molecularly confirmed PK deficiency. Perinatal complications were common, including anemia that required transfusions, hyperbilirubinemia, hydrops, and prematurity. Nearly all newborns were treated with phototherapy (93%), and many were treated with exchange transfusions (46%). Children age 5 years and younger were often transfused until splenectomy. Splenectomy (150 [59%] of 254 patients) was associated with a median increase in hemoglobin of 1.6 g/dL and a decreased transfusion burden in 90% of patients. Predictors of a response to splenectomy included higher presplenectomy hemoglobin (P = .007), lower indirect bilirubin (P = .005), and missense PKLR mutations (P = .0017). Postsplenectomy thrombosis was reported in 11% of patients. The most frequent complications included iron overload (48%) and gallstones (45%), but other complications such as aplastic crises, osteopenia/bone fragility, extramedullary hematopoiesis, postsplenectomy sepsis, pulmonary hypertension, and leg ulcers were not uncommon. Overall, 87 (34%) of 254 patients had both a splenectomy and cholecystectomy. In those who had a splenectomy without simultaneous cholecystectomy, 48% later required a cholecystectomy. Although the risk of complications increases with severity of anemia and a genotype-phenotype relationship was observed, complications were common in all patients with PK deficiency. Diagnostic testing for PK deficiency should be considered in patients with apparent congenital hemolytic anemia and close monitoring for iron overload, gallstones, and other complications is needed regardless of baseline hemoglobin. This trial was registered at www.clinicaltrials.gov as #NCT02053480.

Developing professional caregivers' empathy and emotional competencies through mindfulness-based stress reduction (MBSR): results of two proof-of-concept studies.

OBJECTIVES: To assess the feasibility and acceptability of a mindfulness-based stress reduction (MBSR)-based intervention and determine if the intervention is associated with a significant signal on empathy and emotional competencies. DESIGN: Two pre-post proof-of-concept studies. SETTING: Participants were recruited at the University of Montreal's Psychology Department (Study 1) and the CHU Sainte-Justine Department of Hematology-Oncology (Study 2). PARTICIPANTS: Study 1: 12 students completed the 8-week programme (mean age 24, range 18-34). Study 2: 25 professionals completed the 8-week programme (mean age 48, range 27-63). INTERVENTION: Standard MBSR programme including 8-week mindfulness programme consisting of 8 consecutive weekly 2-hour sessions and a full-day silent retreat. OUTCOMES MEASURES: Mindfulness as measured by the Mindful Attention Awareness Scale; empathy as measured by the Interpersonal Reactivity Index (IRI)'s Perspective Taking and Empathic Concern subscales; identification of one's own emotions and those of others as measured by the Profile of Emotional Competence (PEC)'s Identify my Emotions and Identify Others' Emotions subscales; emotional acceptance as measured by the Acceptance and Action Questionnaire-II (AAQ-II) and the Emotion Regulation Scale (ERQ)'s Expressive Suppression subscale; and recognition of emotions in others as measured by the Geneva Emotion Recognition Test (GERT). RESULTS: In both studies, retention rates (80%-81%) were acceptable. Participants who completed the programme improved on all measures except the PEC's Identify Others' Emotions and the IRI's Empathic Concern (Cohen's d median=0.92, range 45-1.72). In Study 2, favourable effects associated with the programme were maintained over 3 months on the PEC's Identify my Emotions, the AAQ-II, the ERQ's Expressive Suppression and the GERT. CONCLUSIONS: The programme was feasible and acceptable. It was associated with a significant signal on the following outcomes: perspective taking, the identification of one's own emotions and emotional acceptance, thus, justifying moving towards efficacy trials using these outcomes.