Quality of life assessments in a cohort of Mozambican children with sickle cell disease.

INTRODUCTION: sickle cell disease (SCD) has significant pediatric morbidity and mortality in sub-Saharan Africa, where access to therapies such as hydroxyurea and opioids is often limited. Poor disease control and Pain management adversely affects the well-being and mental health of affected children. Questionnaires have been utilized in other regions to report the quality of life (QOL) in children with SCD, but assessments from Africa are lacking. METHODS: children age 2-14 years with SCD presenting for routine outpatient consultations at Hospital Central de Maputo from June-August 2017 were offered participation. After informed consent, the Pediatric QOL Inventory (PedsQL) SCD Module was administered to all caregivers and children > 5 years. Responses were scored from 0-100, with higher scores representing better QOL. RESULTS: a total of 14 children were included, with six (43%), four (29%), two (14%), and two (14%) from the age groups of 2-4, 5-7, 8-12, and 13-14 years, respectively. Mean overall patient QOL was 65.3 and 56.0 in child and caregiver respondents. In patients > 5 years, the difference in mean overall QOL for those on/not on hydroxyurea was 0.6 (66.5-64.9) in child respondents and 15.8 (68.4-52.6) in caregiver respondents. Domains related to worry/emotions and communication scored lower in QOL than Pain-related domains for both patient and caregiver respondents. CONCLUSION: SCD has a negative impact on QOL as reported by this cohort of Mozambican pediatric patients and caregivers, with Pain being less of a concern than emotional and interpersonal issues. A comprehensive, child-focused care approach with robust psychosocial support is needed.

Pediatric hematopoietic cell transplantation: Longitudinal trends in body mass index and outcomes.

Pediatric recipients of HCT may have a high susceptibility for overweight and obesity, and obesity may negatively impact post-transplant mortality and survival. This is a single-center retrospective analysis of 297 pediatric patients who received HCT between 2005 and 2018. Patients were classified as UW, NW, OW, or OB based on age-adjusted BMI. A mixed-effects linear regression model controlling for patient, disease, and transplant-related characteristics was used to trend weight longitudinally. Comparisons were made between weight category and post-transplant outcomes. In the pretransplant period, 5.4%, 54.5%, 22.2%, and 17.8% of patients were UW, NW, OW, and OB, respectively. Five years post-transplantation, those numbers were 10.6%, 48.2%, 16.5%, and 24.7%. Overall, BMI increased 0.00094 ± 0.0001 kg/m2 each day post-transplant (P < .001), with older individuals demonstrating greater rates of increase. Further, there was a larger BMI increase in patients without TBI compared with those who received TBI (1.29 ± 0.49, P = .008). Rates of acute GVHD, chronic GVHD, and viral infections, in addition to time to platelet and neutrophil engraftment and 5-year survival estimates, were not significantly different based on pretransplant BMI. Overweight and obese individuals had poorer 5-year survival based on 100-day post-transplant BMI (P = .02). Overall, pediatric HCT recipients are at risk of developing obesity, which is associated with decreased survival. Adolescents and young adults demonstrate the highest risk of weight gain, representing a vulnerable population that requires close monitoring, additional interventions, and further research.

Successful engraftment of haploidentical bone marrow with post-transplantation cyclophosphamide in patients with aplastic anemia.

Patients with severe aplastic anemia (SAA) may benefit from hematopoietic stem cell transplantation, but many of them lack a matched donor. Haploidentical transplantation is increasingly utilized for the treatment of nonmalignant disease where patients lack a matched donor. We report patients with aplastic anemia who experienced successful engraftments of haploidentical stem cells with post-transplantation cyclophosphamide (PTCy). Case series and review of the literature. We present two cases of pediatric patients with severe aplastic anemia who experienced successful engraftment of haploidentical related bone marrow. Both patients received conditioning consisting of rabbit ATG, cyclophosphamide, fludarabine, and total body irradiation pretransplant, with PTCy. The conditioning regimen was well tolerated by both patients, and they achieved full donor engraftment and were weaned off all immunosuppressants. Haploidentical stem cell transplantation in patients with severe aplastic anemia may be an effective alternative when fully matched donors are not available. PTCy can facilitate successful engraftment and therefore expand the pool of eligible donors for patients with aplastic anemia.