No abstract

ObjectiveChildhood and adolescent cancer can result in high burden of distressing symptoms, particularly in high-risk malignancies. The Symptom Screening in Pediatrics Tool (SSPedi) is a reliable and valid approach to measure bothersome symptoms in paediatric patients receiving cancer treatments. Objective was to describe the feasibility of using SSPedi administration among paediatric patients with high-risk malignancies.MethodsWe conducted a single-centre, cross-sectional study of patients aged 8–18 years with high-risk malignancies in a French paediatric oncology unit. Patients self-reported the degree of bothersome symptoms using SSPedi and difficulty with SSPedi completion. The total SSPedi Score ranging from 0 to 60 (where 60 is worst) and most common moderately bothersome symptoms (scored ≥2 on 0–4 Likert Scale) were described. Feasibility was defined as more than 75% of patients agreeing to participate and more than 90% completion of SSPedi questionnaire.ResultsOut of 16 patients approached, 1 declined participation. Median age was 13 years (IQR 8–19). All were able to self-report SSPedi without difficulty. Patients experienced a median number of 6 (range 0–15) bothersome symptoms (score >0). The mean total SSPedi Score was 12 (SD=9.4). Most common moderately bothersome symptoms were pain (8/15), changes in hunger (8/15) and feeling tired (7/15).ConclusionPatient-reported symptom assessment among children and adolescents with high-risk malignancies is feasible using SSPedi. These patients experience a high burden of bothersome symptoms.

Alpha-thalassemia commonly results from deletions of one (alpha +)or both (alpha zero) of the duplicated α-globin genes on chromosome 16 and, less frequently, from point mutations involving mainly the predominantly expressed α2-globin gene (HBA2). 1 Molecular defects generating unstable α-globin chain variants are part of this category of nondeletional α-thalassemia. Clinical heterogeneity of hemoglobin (Hb) H disease, the common form of clinically significant alpha thalassemia, is mainly related to the genetic background, nondeletional HbH disease being more severe than the deletional forms. 1 In Hb Bart's hydrops foetalis syndrome (BHFS), which associates severe in utero anemia, tissue hypoxia, developmental abnormalities, heart failure, and hydrops fetalis, all α-globin genes are generally missing. Occasionally, hydrops fetalis can result from the inheritance of a nondeletional variant from one parent and an alpha zero thalassemia allele from the other. These so-called "HbH hydrops syndromes" were also described in patients homozygous or compound heterozygous for a severe nondeletional HBA2 variant. 1 Hemoglobin Agrinio (HBA2:c.89T>C) is an α2-globin gene variant leading to a Leucine to Proline substitution at codon 30 of the α-globin chain. This very rare variant, first described in 1993, generates a

No abstract

No abstract