Background: Sickle cell disease (SCD) is one of the most common inherited red blood cell disorders in the world, and the vast majority of infants born with SCD are from Sub-Saharan Africa (SSA). Recent studies have definitively established that hydroxyurea is safe and highly effective for children with SCD in SSA. However, there is a lack of data on the long-term outcomes for children with SCD in SSA beyond early childhood. Procedure: We conducted a retrospective analysis of AYA SCD patients ≥16 yo who received care in a pediatric SCD clinic in Malawi between January 2015 and December 31, 2022 Results: We identified 207 patients ≥16 yo in the SCD clinic electronic database. The vast majority (195, 94%) were alive and receiving care in the pediatric SCD clinic. Three patients had died, and 9 were lost to follow-up. The mean age of patients alive and receiving care was 18±1.9 yo. The mean age at diagnosis was 5.7±1.1 yo. Nearly all AYA patients (N=187, 96%) were taking hydroxyurea for a mean duration of 6±2.7 years. Mean baseline laboratory values were: hemoglobin 8.0±1.3 g/dL, mean corpuscular volume 92±8.4 fl, white blood count 14.2±7.0 × 10 3 /µL, absolute neutrophil count (ANC) 6.2±4.8 × 10 3 /µL, and platelets 343±167 × 10 3 /µL. Among all patients, 21% had at least one transfusion, and 23% of patients had been hospitalized at least once for sickle cell-related complications. Conclusion: We provide the first description of an AYA cohort with SCD receiving routine care with hydroxyurea in a dedicated pediatric clinic under real-world conditions. This study highlights hydroxyurea’s long-term feasibility and underscores the need to enhance the capacity for comprehensive adult care, especially in SSA’s rapidly expanding AYA population.

Objectives This systematic review provides an overview of the effect of malnutrition on the pharmacokinetics of chemotherapy in children with cancer. Methods PubMed, Embase and Cochrane were searched to identify eligible studies. Malnutrition was referred to as undernutrition, as defined by the World Health Organisation and the Gomez Criteria. Results Four studies with a total of 668 children with cancer were included and n=121 (18%) were malnourished. In vincristine, the differences in pharmacokinetic parameters were statistically significant where clearance rates were commonly lower and area under the curve was increased in malnourished children. Conclusion The results are suggestive for pharmacokinetic alterations of chemotherapy in malnourished children with cancer. However, the data is scarce, groups are small, and most studies have been performed in high-income countries. Pharmacokinetic research among (severely) malnourished children with cancer is needed in order to improve their outcome, directed by sub-group and ultimately individualized drug dosing.

Background: Sickle cell disease (SCD) is characterized by both acute and chronic complications that affect the daily lives of patients and lower their quality of life. Objective: To describe the health-related quality of life (HRQoL) and the associated factors in children aged 8 to 17 with SCD attending the paediatric haematology clinic at Kamuzu Central Hospital (KCH) in Lilongwe, Malawi. Methods: A mixed methods cross sectional study was conducted at KCH. Patient data was collected with the aid of a standardized case report form. HRQoL was assessed using PedsQL™ Sickle Cell Disease Module by child’s report. Associations between HRQoL scores and independent variables were evaluated by a linear regression model. In-depth interviews were then carried out and the qualitative data was analyzed using content thematic analysis. Results: A hundred and sixty-three children with SCD were enrolled, 52.1% were females. Their median age was 11.2±2.7 years. The mean global HRQoL score of the children was 62±17.3. The highest scores were in the treatment domain (72.5±15.1) while the lowest scores were in the emotions domain (55.2±28.7). The mean pain score was 58.8±16.3. The factors associated with low HRQoL scores were pain (β-coefficient -6.97 CI (-3.07,-15.58); p value 0.034) and low haemoglobin levels (β-coefficient 2.29 CI (0.65-3.91); p value 0.006 ). Conclusion: The HRQoL of this population is low. Pain and low hemoglobin were significantly associated with low HRQoL scores. A biopsychosocial model of heath care delivery and health campaigns are recommended to improve the HRQoL of the children.

Background: Patient-reported outcomes (PROs) that assess health-related quality of life (HRQoL) are increasingly important components of cancer care and research that have been infrequently used in sub-Saharan Africa (SSA). We aimed to longitudinally measure HRQoL among pediatric lymphoma patients in Malawi. Methods: We administered the Chichewa Pediatric Patient-Reported Outcome Measurement Information System Pediatric (PROMIS)-25 at diagnosis, active treatment, and follow-up among pediatric lymphoma patients in Lilongwe, Malawi. Mean scores were calculated for the six PROMIS-25 HRQoL domains (Mobility, Anxiety, Depressive Symptoms, Fatigue, Peer Relationships, and Pain Interference) using the PROMIS scoring manual. Results: Seventy-five children completed PROMIS-25 surveys at diagnosis, 35 (47%) during active treatment, and 24 (32%) at follow-up. The majority of patients died (n= 37, 49%) or were lost-to-follow-up (n=8, 11%). Most (n=51, 68%) were male, median age was 10 (IQR 8-12), 48 (66%) presented with advanced Stage III/IV, 61 (81%) were diagnosed with Burkitt lymphoma and 14 (19%) had Hodgkin lymphoma. At diagnosis, HRQoL was poor across all domains, except for Peer Relationships. Improvements in HRQoL during active treatment and follow-up exceeded the minimally important difference. Poor Lanksy performance status ≤ 70 and Pain Intensity = 10 at diagnosis were associated with increased mortality risk and worse survival. Conclusions: Our experience suggests incorporating assessments of HRQoL via PROs in oncology care is feasible in SSA, can provide prognostic information, and generates clinically meaningful data to inform supportive care interventions. Further, PROs offer an opportunity to include patient voices and prioritize holistic patient-centered care even in low-resource settings.