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Medication safety with oral antitumour therapeutics in paediatrics (youngAMBORA): A mixed-methods approach towards a
Phyllis Lensker1,2,3,4, Lisa Cuba1,2,3, Katja Gessner2,3
1Pharmacy Department, Universitätsklinikum Erlangen and Friedrich-Alexander-Universität Erlangen-Nürnberg, Erlangen, Germany.
Insights
A new pediatric care program, youngAMBORA, was developed to address challenges with oral antitumour therapeutics (OAT) in children. This program focuses on medication education, application support, and side effect evaluation, improving safety and outcomes for young cancer patients.
Area of Science:
- Oncology
- Pharmacology
- Pediatrics
Background:
- Oral antitumour therapeutics (OAT) offer improved outcomes and convenience but present complex intake challenges.
- Existing clinical pharmacological/pharmaceutical care programs for OAT have shown success in adults (AMBORA trial).
- Comparable care programs are lacking for pediatric patients receiving OAT.
Purpose of the Study:
- To develop a tailored pharmacological/pharmaceutical care program for pediatric patients receiving OAT (youngAMBORA).
- To identify specific demands and educational needs related to OAT in children.
- To enhance medication safety and treatment adherence in pediatric oncology.
Main Methods:
- A parallel mixed-methods approach was employed.
- Quantitative analysis of tumor entities and OAT used in a pediatric cancer center.
- Qualitative survey of patients, caregivers, and healthcare professionals to assess needs and challenges.
Main Results:
- Leukemia and antimetabolites were the most frequent diagnoses and OAT, respectively.
- 81.8% of pediatric patients desired involvement in oral medication education.
- Healthcare professionals identified 'Smell/taste', 'Refusal of intake', and 'Swallowing problems' as more challenging application issues compared to caregivers.
- Two novel side effects ('Skin dryness', 'Taste changes') were identified, not present in the CTCAE library.
Conclusions:
- The youngAMBORA program will incorporate counseling for OAT, child-friendly application support, and systematic side effect evaluation.
- The program will address 17 relevant side effects from patient and caregiver perspectives.
- Age-appropriate information materials will be developed to support pediatric patients and caregivers.
Objective:
Oral antitumour therapeutics (OAT) are increasingly used due to improvements in outcomes and their convenient application. However, complex intake regimens pose several challenges. The randomised AMBORA trial (Medication Safety With Oral Antitumour Drugs) demonstrated highly positive outcomes of a clinical pharmacological/pharmaceutical care program for adults treated with numerous OAT, but comparable concepts in paediatrics are lacking so far.
Methods:
We used a parallel mixed-methods approach to develop a tailored pharmacological/pharmaceutical care program for OAT in paediatrics (youngAMBORA). We combined a quantitative analysis of tumour entities and used OAT in a paediatric cancer centre with a qualitative survey for patients, caregivers, and healthcare professionals to identify particular demands and educational needs (e.g., application problems, side effects).
Results:
Leukaemia (77/315) and antimetabolites (95/151) were the most frequently observed entity and OAT, respectively. Of 22 surveyed patients, 81.8% wanted to be involved in oral medication education. Compared to caregivers, significantly more healthcare professionals graded the three most common application problems to be challenging ('Smell/taste': 32/36 vs. 23/42, p = 0.001; 'Refusal of intake': 31/36 vs. 16/42, p<0.001; 'Swallowing problems': 28/36 vs. 21/42, p = 0.011). We identified nine relevant side effects, of which two ('Skin dryness', 'Taste changes') were not included in 15 previously published core side effects of the Common Terminology Criteria of Adverse Events (CTCAE) item library.
Conclusion:
Based on the present findings, the tailored youngAMBORA care program will include: 1) counselling sessions for classic and targeted OAT, 2) child-friendly support with drug application, and 3) systematic evaluation of 17 relevant side effects from patients' and caregivers' points of view including age-appropriate information material.
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