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. 2018 Dec 18;59(5):625–637. doi: 10.1002/jcph.1359

Table 1.

Summary of Oncology Drugs That Have Received US FDA Approval for Use in Pediatrics Since 2002 and Comparison of Dosing Regimens Between Pediatric and Adult Patients for Specific Indications

Trade Name (Generic Name) Pediatric Labeling Datea Pediatric Indication(s) Pediatric Dosing Regimen and Supporting Evidence Adult Indication(s) Adult Dosing Regimen S/H Was Efficacy in Children Similar to Adults? (Per Approved Label)
Arranon (nelarabine) 10/2005 T‐cell ALL; T‐cell lymphoblastic lymphoma (age range studied: 2.5‐21.7 years) 650 mg/m² IV over 1 hour daily for 5 consecutive days repeated every 21 days; based on 1 clinical trial in pediatric patients T‐cell ALL and T‐cell lymphoblastic lymphoma 1500 mg/m² IV over 2 hours on days 1, 3, and 5 repeated every 21 days H Yes; based on complete response data
Afinitor (everolimus) 10/2010 and 8/2012 Subependymal giant cell astrocytoma (SEGA) associated with tuberous sclerosis in patients ≥3 years of age 4.5 mg/m2 orally QD; adjust dose to attain trough concentrations of 5‐15 ng/mL; based on 1 clinical trial in pediatric Advanced hormone receptor‐positive, HER2‐negative breast cancer; progressive neuroendocrine tumors; advanced renal cell carcinoma; renal angiomyolipoma and tuberous sclerosis complex; SEGA 10 mg orally QD S Yes; for SEGA based on 1 trial with pediatrics and young adults patients
Bavencio (avelumab) 3/2017 Metastatic MCC in patients aged ≥12 years 10 mg/kg IV over 60 minutes every 2 weeks; based on several clinical trials in adult patients and population pharmacokinetics data to support use in pediatric patients aged ≥12 years Metastatic MCC Same as pediatric S Yes; based on full extrapolation from adults using population pharmacokinetics data and assuming the course of MCC is sufficiently similar in adults and pediatric patients
Blincyto (blinatumomab) 8/2016 B‐cell precursor ALL in first or second complete remission with MRD ≥0.1%; R/R B‐cell precursor ALL (age range studied: <1‐17 years)

  • Cycle 1 dosing (all doses IV)

  • MRD positive:

  • Weight ≥45 kg: 28 μg/day for 28 days

  • Weight <45 kg: 15/μg/m2/day, not to exceed 28 μg/day, for 28 days

  • R/R:

  • Weight ≥45 kg: 9 μg/day for 7 days, 28 μg/day for 21 days

  • Weight <45 kg: 5/μg/m2/day, not to exceed 9 μg/day, for 7 days, 15 μg/m2/day, not to exceed 28 μg/day, for 21 days

  • Based on limited experience in 41 pediatric patients in a dose‐escalation study

 B‐cell precursor ALL in first or second complete remission with MRD ≥0.1% or R/R B‐cell precursor ALL Based on weight; same as pediatric H Yes; based on partial extrapolation from adults with MRD‐positive B‐cell precursor ALL
Busulfex (busulfan) 1/2003 Part of a conditioning regimen in hematopoietic stem cell transplant (age range studied: 5 months to 16 years)

  • Weight ≤12 kg: 1.1 mg/kg IV

  • Weight > 12 kg: 0.8 mg/kg IV;

  • Based on pharmacokinetics/tolerability study and extrapolation from adults CML data

 Part of a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplant for CML 0.8 mg/kg of ideal body weight or actual body weight as a 2‐hour IV infusion every 6 hours for 4 consecutive days for a total of 16 doses H Yes; based on full extrapolation from adult CML
Clolar (clofarabine) 12/2004 R/R ALL in patients aged 1‐21 years 52 mg/m2 IV over 2 hours daily for 5 consecutive days of a 28‐day cycle; based on one clinical trial in R/R ALL in pediatrics and young adults NA NA H Yes; based on data in pediatrics and young adults in R/R ALL
Erwinaze (asparaginase Erwinia chryanthemi) 11/2011 ALL in patients with hypersensitivity to E coli‐derived asparaginase (age range studied: 2–18, 1‐17, and 0‐76 years) 25,000 IU/m2 IM or IV 3 times a week; based on 2 clinical trials in pediatric patients NA NA H Efficacy not established in adults
Gleevec (imatinib mesylate) 9/2006 Newly diagnosed Ph+ CML in chronic phase; Ph+ acute ALL (age range studied: ≥1 year) 340 mg/m2/day orally QD (not to exceed 600 mg) for Ph+ CML or Ph+ ALL or as a split dose in the morning and evening for Ph+ CML; based on several clinical trials in pediatric patients Ph+ CML; Ph+ ALL; myelodysplastic/myeloproliferative diseases; aggressive systemic mastocytosis; hypereosinophilic syndrome and/or chronic eosinophilic leukemia; dermatofibrosarcoma protuberans; malignant gastrointestinal stromal tumors 400‐800 mg orally QD depending upon the indication H Yes; based on complete cytogenic response rate for Ph+ CML in chronic phase. Ph+ acute ALL used a different end point relative to adults
Keytruda (pembrolizumab) 11/2017 Refractory cHL; refractory PMBCL; unresectable or metastatic MSI‐H or mismatch repair deficient solid tumors; colorectal cancer (age range studied: 2‐18 years) 2 mg/kg (up to 200 mg) IV every 3 weeks; based on limited clinical studies in pediatric patients and extrapolation from several studies in adults Melanoma; NSCLC; HNSCC; classical Hodgkin lymphoma, PMBCL; urothelial carcinoma; microsatellite instability‐high cancer; gastric cancer; cervical cancer 200 mg IV over 30 minutes every 3 weeks S/H Yes; based on full extrapolation from adults data in cHL, PMBCL and MSI‐H
Kymriah (tisagenlecleucel) 8/2017 B‐cell precursor ALL refractory or in second or later relapse in patients aged ≤25 years

  • Weight ≤50 kg: 0.2‐5.0 × 106 CAR‐positive viable T cells per kg body weight administered IV

  • Weight >50 kg: 0.1‐2.5 × 108 total CAR‐positive viable T cells (non–weight based) administered IV;

  • based on 1 clinical trial in pediatrics and young adults r/r B‐cell precursor ALL

 B‐cell ALL (young adults); R/R DLBCL after 2+ lines of systemic therapy 0.6 to 6.0 × 108 CAR‐positive viable T cells administered IV (R/R DLBCL) H Yes; based on CR rate in r/r B‐cell ALL
Mylotarg (gemtuzumab ozogamicin) 9/2017 R/R CD33+ AML in patients aged ≥2 years 3 mg/m2 (up to one 4.5 mg vial) IV on days 1, 4, and 7; based on 1 clinical trial in pediatric r/r AML patients Newly diagnosed or R/R CD33+ AML 3 mg/m2 (up to one 4.5 mg vial) IV on days 1, 4, and 7 in combination with daunorubicin and cytarabine; 6 mg/m2 IV on day 1 and 3 mg/m2 on day 8 as a single agent H Yes; based on CR rates in r/r AML pediatric and young adults
Sprycel (dasatinib) 11/2017 Ph+ CML in chronic phase (age range studied: ≥1 year) Starting dose of 40‐100 mg orally QD based on body weight; tablet dosing not recommended in patients weighing <10 kg; based on 2 clinical trials in pediatric patients with chronic‐phase CML Newly diagnosed Ph+ CML in chronic phase; chronic, accelerated, or myeloid or lymphoid blast phase Ph+ CML resistant or intolerant to prior therapy including imatinib; Ph+ chromosome‐positive ALL with resistance or intolerance to priory therapy Starting dose of 100‐140 mg orally QD H Higher efficacy in pediatrics based on comparison of complete cytogenetic response rates within 12 months
Tasigna (nilotinib) 3/2018 Newly diagnosed Ph+ CML in chronic phase or newly diagnosed Ph+ CML in chronic phase resistant or intolerant to prior TKI therapy in patients aged ≥1 year 230 mg/m2 orally BID, rounded to the nearest 50‐mg dose (to a maximum single dose of 400 mg); based on two clinical trials in pediatric patients Newly diagnosed Ph+ CML in chronic phase; chronic phase and accelerated phase Ph+ CML resistant/intolerant to prior therapy including imatinib 300‐400 mg orally BID H Yes; for newly diagnosed Ph+ CML
Unituxin (dinutuximab) 3/2015 High‐risk neuroblastoma (in combination with with GM‐CSF, IL‐2, and 13‐cis‐retinoic acid) (age range studied: 11 months to 15 years) 17.5 mg/m2/day IV over 10‐20 hours for 4 consecutive days for up to 5 cycles; based on 1 clinical trial in pediatric patients NA NA S Efficacy not established in adults; neuroblastoma is a pediatric‐specific cancer
Xgeva (denosumab) 6/2013 Giant cell tumor of bone in skeletally mature adolescents (age range studied: 13‐17 years) 120 mg SC every 4 weeks with additional 120 mg doses on days 8 and 15 of the first month of therapy; based on one clinical trial in adolescent patients. Prevention of skeletal‐related events in patients with MM and patients with bone metastases from solid tumors; giant cell tumor of bone 120 mg SC every 4 weeks; 120 mg SC every 4 weeks with additional 120 mg doses on days 8 and 15 of the first month of therapy for patients with giant cell tumor of bone S Yes; based on efficacy in skeletally mature adolescents and adults

ALL, acute lymphoblastic leukemia; AML, acute myelogenous leukemia; BID, twice daily; CAR, chimeric antigen receptor; cHL, classical Hodgkin lymphoma; CML, chronic myelogenous leukemia; DLBCL, diffuse large B‐cell lymphoma; GM‐CSF, granulocyte‐macrophage colony stimulating factor; HER2, human epidermal growth factor receptor 2; HNSCC; head and neck squamous cell carcinoma; IL‐2, interleukin‐2; IM, intramuscular; IV, intravenous; MCC, Merkel cell carcinoma; MM, multiple myeloma; MRD, minimal residual disease; MSI‐H, microsatellite instability‐high; NA, not applicable; NSCLC, non–small cell lung cancer; Ph +, Philadelphia chromosome positive; PMBCL, primary mediastinal B‐cell lymphoma; QD, once daily; R/R, relapsed or refractory; SC, subcutaneous; S/H, solid tumor or hematological malignancy; TKI, tyrosine kinase inhibitor.

a

Based on US FDA approvals obtained since 2002 (adapted from FDA2,3). The indications and dosing regimens have been summarized for brevity; consult approved labeling for complete descriptions of indications and dosing regimens.