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.

medium11 referencesUpdated 17 July 202620 min readVerification in progress

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RACP General PaediatricsRACP DWERACP DCERCPCH Progress+MRCPCH TheoryMRCPCH ClinicalABP General PediatricsACGME PediatricsRCPSC Pediatrics

Red flags

Vincristine prepared or delivered by the intrathecal route — a uniformly fatal administration error that must never occurAn anthracycline such as doxorubicin or daunorubicin given without a documented cumulative-dose record and cardiac surveillance planAnthracycline extravasation managed with cold compresses rather than dexrazoxane — cold worsens vinca injury and cold is wrong for anthracyclineOndansetron given in large intravenous doses without awareness of dose-related QT prolongationA child on chemotherapy presenting with febrile neutropenia treated as a routine viral illness rather than a medical emergencyDexrazoxane withheld because of an out-of-date fear of secondary malignancy when the child is receiving a high cumulative anthracycline dose

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infanttoddlerpreschoolschool-ageadolescentyoung-adult-transition

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General and Community PaediatricsAllergy and ImmunologyCurrent 2026 PREP curriculum — Learning Objective: Safe prescribing and medication management in infants, children and young peopleCurrent 2026 PREP curriculum — Learning Objective 2.2.1: Recognise, prioritise and manage an acutely ill infant, child or young personRenewed curriculum for first-year trainees from 2027 — Learning goal 6: Clinical management – essential general paediatricsRenewed curriculum for first-year trainees from 2027 — Learning goal 9: Quality and safety, including medication safetyClinical ApplicationsMedical SciencesLong CasesShort Cases4. Professional skills and knowledge: Patient management7. Patient safety, including safe prescribing8. Quality improvement and patient safetyGeneral Paediatrics: Understands cytotoxic chemotherapy classes, supportive care medicines, and the non-negotiable vincristine-intrathecal safety ruleFoundation of Practice (FOP)Theory and Science (TAS)Applied Knowledge in Practice (AKP)ClinicalHistoryCommunicationGeneral Pediatrics Content Outline — Domain 10: Principles of Pharmacology and Medication ManagementGeneral Pediatrics Content Outline — Universal Task 4: Management and TreatmentGeneral Pediatrics Content Outline — Universal Task 2: Health MaintenanceSystems-Based Practice 2: Coordinated chronic disease carePatient Safety/Quality Improvement 1: Medication SafetyMedical Knowledge 2: Diagnostic and Therapeutic Decision MakingMedical ExpertCollaboratorPediatrics: Core EPA #5 — Recommending and Initiating Pharmacologic Interventions

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Target exams

RACP General PaediatricsRACP DWERACP DCERCPCH Progress+MRCPCH TheoryMRCPCH ClinicalABP General PediatricsACGME PediatricsRCPSC Pediatrics

Red flags

Life stages

infanttoddlerpreschoolschool-ageadolescentyoung-adult-transition

Care settings

outpatientwarded-acutepicutelehealth

Clinical exam formats

written-only

Board mappings

The fellowship answer

Chemotherapy and supportive pharmacology in children pair each cytotoxic drug class with the supportive medicine that prevents its characteristic harm. The anthracyclines doxorubicin and daunorubicin kill cancer cells but injure the myocardium, and dexrazoxane — an intracellular iron chelator given before the anthracycline — protects the heart and also rescues anthracycline extravasation. The vinca alkaloids block microtubules and cause neurotoxicity, and vincristine must travel by the intravenous route only — into the cerebrospinal fluid it is uniformly fatal, the single most dreaded chemotherapy administration error. Chemotherapy-induced nausea and vomiting rise on a ladder: the 5-HT3 antagonist ondansetron, the NK1 antagonist aprepitant, and dexamethasone are combined for the highly emetogenic regimens. Febrile neutropenia, the great killer of the myelosuppressed child, is prevented with the colony-stimulating factors filgrastim and the long-acting pegfilgrastim. And the severe oral mucositis that follows stem-cell transplant conditioning is reduced by palifermin, a keratinocyte growth factor. State the toxicity, name its antidote, and protect the route — that is the spine of safe paediatric oncology pharmacology.

[1][6][9]

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 · 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 · Match the supportive medicine

Pair the toxicity with its antidote — dexrazoxane, antiemetics, colony-stimulating factors, palifermin, hydration and rasburicase as indicated.

3 · Protect the route

Confirm vincristine is intravenous only and never prepared with intrathecal drugs; apply independent checks and minibag dilution.

4 · Plan the surveillance

Track cumulative anthracycline dose with echocardiography and troponin, blood counts for neutropenia, and growth and development throughout.

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.

[6] [7]

Paediatrics Fellowship Pro

You have read the opening of this topic. The complete unit — every section and its primary-source references — is part of the Paediatrics Fellowship fellowship atlas.

References11Show ledger

[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

Chemotherapy and supportive pharmacology

medium11 referencesUpdated 17 July 202620 min readVerification in progress

Practise this topic

Overview & Definition

From the cytotoxic plan to the supported child

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 · Match the supportive medicine

Pair the toxicity with its antidote — dexrazoxane, antiemetics, colony-stimulating factors, palifermin, hydration and rasburicase as indicated.

3 · Protect the route

Confirm vincristine is intravenous only and never prepared with intrathecal drugs; apply independent checks and minibag dilution.

4 · Plan the surveillance

Track cumulative anthracycline dose with echocardiography and troponin, blood counts for neutropenia, and growth and development throughout.

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.

[6]

[7]

Paediatrics Fellowship Pro

You have read the opening of this topic. The complete unit — every section and its primary-source references — is part of the Paediatrics Fellowship fellowship atlas.

References11Show ledger

[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