Children with sickle cell disease (SCD) demonstrate cerebral hemodynamic stress and are at high risk of strokes. We hypothesized that curative hematopoietic stem cell transplant (HSCT) normalizes cerebral hemodynamics in children with SCD compared to pre-transplant baseline. Whole-brain cerebral blood flow (CBF) and oxygen extraction fraction (OEF) were measured by MRI 1-3 months before and 12-24 months after HSCT in ten children with SCD: three with prior overt strokes, five with prior silent strokes, and one with abnormal transcranial Doppler ultrasound velocities. HSCT recipients' CBF and OEF were compared to non-SCD controls and SCD participants receiving chronic red blood cell transfusion therapy (CRTT) before and after a scheduled transfusion. Seven participants received matched sibling donor HSCT; three received 8/8 matched unrelated donor HSCT. All received reduced-intensity preparation and maintained engraftment, free of hemolytic anemia and SCD symptoms. Pre-transplant, CBF (93.5 ml/100g/min) and OEF (36.8%) were elevated compared with non-SCD controls, declining significantly 1-2 years following HSCT (CBF 72.7 ml/100g/min, p=0.004; OEF 27.0%, p=0.002), with post-HSCT CBF and OEF similar to non-SCD control subjects. Furthermore, HSCT recipients demonstrated greater reduction in CBF (-19.4 ml/100g/min) and OEF (-8.1%) after HSCT than children with SCD receiving CRTT after a scheduled transfusion (CBF -0.9 ml/100g/min, p=0.024; OEF -3.3%, p=0.001). HSCT normalizes whole-brain hemodynamics following curative HSCT in children with SCD. This restoration of cerebral oxygen reserve may explain stroke protection following HSCT in this high-risk patient population.

PEG-aspariginase is a backbone chemotherapy agent in pediatric acute lymphoblastic leukemia and in some non-Hodgkin lymphoma therapies. Nurses lack standardized guidelines for monitoring patients receiving PEG-asparaginase and for educating patients/families about hypersensitivity reaction risks. An electronic search of six databases using publication years 2000–2015 and multiple professional organizations and clinical resources was conducted. Evidence sources were reviewed for topic applicability. Each of the final 23 sources was appraised by two team members. The GRADE system was used to assign a quality and strength rating for each recommendation. Multiple recommendations were developed: four relating to nurse monitoring of patients during and after drug administration, eight guiding hypersensitivity reaction management, and four concerning patient/family educational content. These strong recommendations were based on moderate, low, or very-low quality evidence. Several recommendations relied upon generalized drug hypersensitivity guidelines. Additional research is needed to safely guide PEG-asparaginase monitoring, hypersensitivity reaction management and patient/family education. Nurses administering PEG-asparaginase play a critical role in the early identification and management of hypersensitivity reactions.

Background: Sickle cell disease (SCD) may cause injury to any organ, including the auditory system. Although the association of SCD and hearing loss has been described, the nature of this complication is unknown. We sought to establish the prevalence and nature of hearing loss in a referred cohort of children with SCD and to identify correlating disease-or treatment-associated factors. Procedure:We conducted a retrospective review of patients with SCD < 22 years of age who had hearing evaluations between August 1990 and December 2014. Demographics, audiograms, and disease and treatment variables were analyzed.Results: Two hundred and ten audiograms among 81 patients were reviewed, and 189 were evaluable. Seventy-two children constituted the referred cohort. Fourteen (19.4%) had hearing loss documented on at least one audiogram. Seven (9.7%) patients had only conductive hearing loss, and the loss persisted for up to 10.3 years. The median age of first identification was eight years.Six (8.3%) patients had hearing loss that was at least partially sensorineural. One patient's hearing loss was ambiguous. All sensorineural hearing losses were unilateral and 4/6 patients had prior documented normal hearing, indicating acquired loss. No correlations were identified. Conclusions:Both conductive and sensorineural hearing losses are more prevalent in our study population than those observed in the general pediatric population. In children with SCD, sensorineural hearing loss appears to be acquired and unilateral. Conductive hearing loss was identified in older children and can persist. Serial screening is needed for early detection and more prompt intervention in this population.

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Hematopoietic cell transplantation (HCT) is an elective, curative treatment option for patients with sickle cell disease (SCD). Transplant requires extensive self-management behaviors to be successful. The purpose of this study was to describe potential barriers and facilitators to self-management in a group of pediatric patients with SCD prior to HCT and their medical outcomes post-HCT. A multiple case study approach was used to describe 4 pairs of transplant recipients grouped by age, donor type, and donor source. Each pair included a case with minimal and increased post-HCT complications. Complications included readmissions, graft-versus-host disease, systemic infections, and survival in the first year post-HCT. Variables were retrospectively collected and content analyzed to identify barriers and facilitators within and across pairs using existing self-management frameworks. While higher risk transplants experienced more complications, 3 of the 4 cases with increased complications had a larger number of modifiable barriers identified compared with those experiencing minimal complications. At least one modifiable barrier and multiple facilitators were identified in all cases. A standardized psychosocial assessment process with an established plan to mitigate barriers and promote facilitators to self-management is essential to optimize outcomes in patients with SCD undergoing elective HCT.