(251) The real-world use of medicines for the management of haematological malignancies in paediatric populations: an international scoping review.

Background: Understanding real-world pharmacological interventions for paediatric patients diagnosed with haematological malignancies is necessary considering the burden of disease and current uncertainty around actual clinical care to direct improvement in prescribing medicines and hence enhancing patients’ experiences.

Objectives: To identify prescribed systematic anti-cancer therapy (SACT) along with supplementary medicines for paediatric patients who accessed clinical facilities seeking the management of haematological malignancies in real-world circumstances.

Methods: A scoping review was conducted using three relevant databases namely, PubMed (Medline), Embase, Scopus in accordance with Joanna Briggs Institute's (JBI) framework. Grey literature and reference lists of selected articles were also checked for unpublished relevant data. Eligible articles were all English-language observational studies of paediatric patients ( < 19 years) with cancer from January 2013 until February 2023 which highlighted the use of medicines for cancer itself and concurrent ailments. Selecting articles began with two-stage screening of retrieved articles. Screening at both stages (title/abstract, and full text) were validated by another researcher. The next stage was the extraction of relevant data. However, during the extraction stage the focus was narrowed to the most common types of cancer (haematological malignancies) among this population due to time restriction.Individual study findings were synthesised narratively.

Results: Of 11899 imported articles, 73 articles were focused on haematological malignancies. Commonly used SACT were methotrexate, asparaginase, vincristine, 6-mercaptupurine, cytarabine, cyclophosphamide and doxorubicin. Using of steroids was also documented along with SACT. The addition of relatively novel medicines such as tisagenlecleucel and brentuximab vedotin was mostly for specific cases; for example, relapsed and refractory cases. Forty-four articles mentioned the use of certain supplementary medicines, such as prophylactic co-trimoxazole against Pneumocystis jiroveci. There were commonalities amongst treatment in developed and developing countries.

Conclusions: This review summarised medicines used in the treatment of paediatric haematological cancers in the real-world observational studies internationally over the last decade. It would be beneficial to elaborate on medicines’ use for other potential concurrent clinical conditions such as insomnia. Further understanding of the extent of unlicenced prescribing and variation in practice would be worthwhile.