Cell therapy in sarcoma: current landscape and future directions
The data demonstrate that matching the donor type, timing of relapse after HSCT, and disease burden at lymphodepletion predict outcomes for tisagenlecleucel in post‑transplant B‑ALL.
Acute lymphoblastic leukaemia (ALL) is one of the most treatable forms of paediatric cancer; however, there is a substantial burden of treatment‑related toxicities (TRTs). The long‑term changes in children’s health‑related quality of life (HRQoL) due to toxic treatments are not well understood. To describe parent‑reported HRQoL of children with ALL over a 5‑year period from diagnosis and identify risk factors associated with parent reporting of low HRQoL in children, we conducted a national longitudinal prospective study. Parents of children diagnosed with ALL within the last 100 days were invited to report the HRQoL impacts on their child over time using the PedsQL Cancer Module. Mixed models identified predictors of HRQoL. A total of 190 parents participated at one or more time points (children’s mean age 6.2 years). Parents reported clinically meaningful reductions in their child’s HRQoL throughout ALL treatment: within the first 6 months of diagnosis, 59.2% reported reduced HRQoL and 47.6% at 54 months. Procedural anxiety and cognitive dysfunction were key contributors to HRQoL throughout and beyond treatment. Age at diagnosis, sex and rurality were associated with HRQoL. Almost half of parents reported that their children experienced poor HRQoL during and after treatment. Procedural anxiety and cognitive dysfunction were key contributors that did not reduce with time, demonstrating the need for comprehensive assessment of children’s HRQoL to support access to psychosocial supportive care needs and improve overall HRQoL into survivorship years.
These findings inform clinical decision‑making regarding CAR‑T therapy in patients who have relapsed after HSCT, highlighting the need for individualized risk assessment to optimise treatment strategies.
Evidence level: Korai humán adat. Kis vagy feltáró emberi adat.
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