Paeds · clinical-pharmacology-and-therapeutics
Chemotherapy and supportive pharmacology
Also known as Cytotoxic chemotherapy in children · Antiemetics in paediatric oncology · G-CSF and pegfilgrastim in children · Dexrazoxane cardioprotection · Palifermin for mucositis · Anthracycline extravasation management
A fellowship approach to chemotherapy and supportive pharmacology in children covering the cytotoxic drug classes and their organ-specific toxicities, the antiemetic ladder built on 5-HT3 antagonists (ondansetron), the NK1 antagonist aprepitant and dexamethasone for chemotherapy-induced nausea and vomiting, the colony-stimulating factors filgrastim and pegfilgrastim for febrile neutropenia prophylaxis, palifermin (keratinocyte growth factor) for oral mucositis after stem-cell transplant conditioning, dexrazoxane for anthracycline cardioprotection and extravasation, and the non-negotiable rule that vincristine is given intravenously and never by the intrathecal route.
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Red flags
- Vincristine prepared or delivered by the intrathecal route — a uniformly fatal administration error that must never occur
- An anthracycline such as doxorubicin or daunorubicin given without a documented cumulative-dose record and cardiac surveillance plan
- Anthracycline extravasation managed with cold compresses rather than dexrazoxane — cold worsens vinca injury and cold is wrong for anthracycline
- Ondansetron given in large intravenous doses without awareness of dose-related QT prolongation
- A child on chemotherapy presenting with febrile neutropenia treated as a routine viral illness rather than a medical emergency
- Dexrazoxane withheld because of an out-of-date fear of secondary malignancy when the child is receiving a high cumulative anthracycline dose
Life stages
Care settings
Clinical exam formats
Board mappings
- General and Community Paediatrics
- Allergy and Immunology
- Current 2026 PREP curriculum — Learning Objective: Safe prescribing and medication management in infants, children and young people
- Current 2026 PREP curriculum — Learning Objective 2.2.1: Recognise, prioritise and manage an acutely ill infant, child or young person
- Renewed curriculum for first-year trainees from 2027 — Learning goal 6: Clinical management – essential general paediatrics
- Renewed curriculum for first-year trainees from 2027 — Learning goal 9: Quality and safety, including medication safety
- Clinical Applications
- Medical Sciences
- Long Cases
- Short Cases
- 4. Professional skills and knowledge: Patient management
- 7. Patient safety, including safe prescribing
- 8. Quality improvement and patient safety
- General Paediatrics: Understands cytotoxic chemotherapy classes, supportive care medicines, and the non-negotiable vincristine-intrathecal safety rule
- Foundation of Practice (FOP)
- Theory and Science (TAS)
- Applied Knowledge in Practice (AKP)
- Clinical
- History
- Communication
- General Pediatrics Content Outline — Domain 10: Principles of Pharmacology and Medication Management
- General Pediatrics Content Outline — Universal Task 4: Management and Treatment
- General Pediatrics Content Outline — Universal Task 2: Health Maintenance
- Systems-Based Practice 2: Coordinated chronic disease care
- Patient Safety/Quality Improvement 1: Medication Safety
- Medical Knowledge 2: Diagnostic and Therapeutic Decision Making
- Medical Expert
- Collaborator
- Pediatrics: Core EPA #5 — Recommending and Initiating Pharmacologic Interventions
Overview & Definition
Picture an eight-year-old receiving doxorubicin for acute lymphoblastic leukaemia whose echocardiogram is tracked cycle by cycle, a six-year-old on cisplatin who vomits for three days after each infusion, and a ten-year-old the morning after stem-cell transplant conditioning whose mouth is so sore he cannot swallow his own saliva. Each child is living a cytotoxic toxicity, and each one has a supportive medicine built to prevent or treat it. The general paediatrician rarely prescribes the cytotoxic agent, but shares the surveillance, the febrile-neutropenia front door, and the safety culture that keeps the routes right. [1] [11]
Two ideas carry the whole topic. The first is the toxicity-to-antidote pairing: every cytotoxic class has a predictable organ injury, and modern paediatric oncology pairs each one with a supportive drug that blunts the injury without abolishing the anti-cancer effect. The second is the route-safety culture: the cytotoxics are among the most dangerous drugs to administer, and the vincristine-intrathecal catastrophe is the cautionary tale that drives minibag dilution, separate intrathecal preparation times, and double-independent checks. [9] [11]
From the cytotoxic plan to the supported child
- 1
1 · Know the drug and its toxicity
Identify the cytotoxic class and the organ it injures — anthracycline to the heart, vinca to nerves, platinum to ears and kidneys, antimetabolite to marrow.
- 2
2 · Match the supportive medicine
Pair the toxicity with its antidote — dexrazoxane, antiemetics, colony-stimulating factors, palifermin, hydration and rasburicase as indicated.
- 3
3 · Protect the route
Confirm vincristine is intravenous only and never prepared with intrathecal drugs; apply independent checks and minibag dilution.
- 4
4 · Plan the surveillance
Track cumulative anthracycline dose with echocardiography and troponin, blood counts for neutropenia, and growth and development throughout.
- 5
5 · Hold the front door
Treat the febrile neutropenic child as a medical emergency: cultures and empirical antibiotics within the hour, never as a routine viral illness.
References11ShowHide
- [1]Lipshultz SE; Rifai N; Dalton VM; Levy DE; Silverman LB; Lipsitz SR The effect of dexrazoxane on myocardial injury in doxorubicin-treated children with acute lymphoblastic leukemia The New England journal of medicine, 2004.PMID 15247354
- [2]Barry EV; Vrooman LM; Dahlberg SE; Neuberg DS; Asselin BL; Athale UH Absence of secondary malignant neoplasms in children with high-risk acute lymphoblastic leukemia treated with dexrazoxane Journal of clinical oncology, 2008.PMID 18309945
- [3]Lipshultz SE; Miller TL; Scully RE; Lipsitz SR; Rifai N; Silverman LB Changes in cardiac biomarkers during doxorubicin treatment of pediatric patients with high-risk acute lymphoblastic leukemia: associations with long-term echocardiographic outcomes Journal of clinical oncology, 2012.PMID 22370326
- [4]Spielberger R; Stiff P; Bensinger W; Gentile T; Weisdorf D; Kewalramani T Palifermin for oral mucositis after intensive therapy for hematologic cancers The New England journal of medicine, 2004.PMID 15602019
- [5]Kang HJ; Loftus S; Taylor A; DiCristina C; Green S; Zwaan CM Aprepitant for the prevention of chemotherapy-induced nausea and vomiting in children: a randomised, double-blind, phase 3 trial The Lancet. Oncology, 2015.PMID 25770814
- [6]Dupuis LL; Sung L; Molassiotis A; Orsey AD; Tissing W; van de Wetering M 2016 updated MASCC/ESMO consensus recommendations: Prevention of acute chemotherapy-induced nausea and vomiting in children Supportive care in cancer, 2017.PMID 27565788
- [7]Smith TJ; Khatcheressian J; Lyman GH; Ozer H; Armitage JO; Balducci L 2006 update of recommendations for the use of white blood cell growth factors: an evidence-based clinical practice guideline Journal of clinical oncology, 2006.PMID 16682719
- [8]Spunt SL; Irving H; Frost J; Sender L; Guo M; Yang BB Phase II, randomized, open-label study of pegfilgrastim-supported VDC/IE chemotherapy in pediatric sarcoma patients Journal of clinical oncology, 2010.PMID 20142595
- [9]Mouridsen HT; Langer SW; Buter J; Eidtmann H; Rosti G; de Wit M Treatment of anthracycline extravasation with Savene (dexrazoxane): results from two prospective clinical multicentre studies Annals of oncology, 2007.PMID 17185744
- [10]van Dalen EC; Caron HN; Dickinson HO; Kremer LC Cardioprotective interventions for cancer patients receiving anthracyclines Cochrane database of systematic reviews, 2011.PMID 21678342
- [11]DeVine A; Landier W; Hudson MM; Constine LS; Bhatia S; Armenian SH The Children's Oncology Group Long-Term Follow-Up Guidelines for Survivors of Childhood, Adolescent, and Young Adult Cancers: A Review JAMA oncology, 2025.PMID 39976936