Most Common Pediatric Malignancies (U.S. / Canada)
- Leukemias (≈30%)
- Acute lymphoblastic leukemia (ALL) → most common overall pediatric cancer
- Acute myeloid leukemia (AML) → less common but more aggressive
- Brain and CNS tumors (≈20%)
- Lymphomas (≈15%)
- Neuroblastoma (≈7%) → most common extracranial solid tumor in children
- Wilms tumor (nephroblastoma) (≈6%) → most common renal malignancy in kids
- Bone tumors
- Other embryonal tumors
- Rhabdomyosarcoma (soft tissue sarcoma)
- Retinoblastoma
1. Epidemiology & Presentation
- ALL = most common childhood cancer
- CNS tumors = most common solid tumor in children
- Neuroblastoma = most common extracranial solid tumor
- Wilms = most common renal tumor in children
- Osteosarcoma = most common bone tumor
- Rhabdomyosarcoma = most common soft-tissue sarcoma in children
2. First-Line Therapy
- ALL: multi-agent chemo (vincristine, corticosteroid, asparaginase, ± anthracycline), maintenance with 6-mercaptopurine + methotrexate.
- AML: cytarabine + anthracycline; stem cell transplant in high-risk.
- CNS tumors: surgery + radiation (limited in very young kids) + chemo.
- Neuroblastoma: risk-adapted chemo, surgery, radiation, immunotherapy (anti-GD2 mAbs).
- Wilms tumor: nephrectomy + chemo (vincristine + dactinomycin ± doxorubicin).
3. Supportive Care / Pharmacist-Specific Points
- Tumor lysis syndrome (TLS): common in ALL, Burkitt lymphoma → recognize labs (↑uric acid, ↑K, ↑Phos, ↓Ca) and prophylaxis (hydration, allopurinol, rasburicase).
- CNS prophylaxis in ALL: intrathecal MTX ± cytarabine ± hydrocortisone.
- Asparaginase toxicities: pancreatitis, thrombosis, hypersensitivity, hyperglycemia → exam favorite.
- Anthracyclines: cardiotoxicity monitoring (echo, EF).
- Vincristine: dose capped at 2 mg, neurotoxicity (never intrathecal!).
Pediatric Brain Tumors
1. Medulloblastoma Definition: Primitive neuroectodermal tumor (PNET) of the cerebellum. Highly malignant and fast-growing.Epidemiology:
Location: Posterior fossa (cerebellar vermis).
Clinical Features:
- Headache, vomiting (due to raised ICP)
- Ataxia, gait disturbances
- Hydrocephalus possible
Treatment & Pharmacist Role:
- Surgery – maximal safe resection.
- Radiotherapy – craniospinal irradiation (post-op).
- Chemotherapy – often cisplatin, vincristine, cyclophosphamide:
- Monitor for nephrotoxicity (cisplatin), neuropathy (vincristine), and myelosuppression (cyclophosphamide).
- Supportive care: hydration, antiemetics, growth factor support if needed.
Pharmacist Notes:
- Dose adjustments may be required for renal dysfunction (cisplatin).
- Be alert for drug-drug interactions with anti-seizure or antiemetic therapy.
- Long-term monitoring: ototoxicity, neurocognitive effects.
2. Astrocytoma (Pediatric) Definition: Tumor derived from astrocytes. Includes low-grade (pilocytic) and high-grade (anaplastic/glioblastoma) variants.
Epidemiology:
- Most common benign/low-grade pediatric brain tumor.
- Peak incidence: 5–10 years.
Location:
- Often cerebellum (posterior fossa) for low-grade
- High-grade may occur in supratentorial region.
Clinical Features:
- Headache, vomiting, visual changes
- Seizures possible if supratentorial
Treatment & Pharmacist Role:
- Surgery – resection often curative for low-grade.
- Radiotherapy – for high-grade or residual disease.
- Chemotherapy – mainly for inoperable or recurrent tumors:
- Carboplatin, vincristine, temozolomide.
- Monitor for myelosuppression, neuropathy, renal function.
Pharmacist Notes:
- Low-grade tumors may require minimal chemo, so supportive care focus.
- High-grade tumors require aggressive chemo-radiation, monitoring for toxicity and neurocognitive late effects.
3. Ependymoma Definition: Tumor of ependymal cells lining the ventricles and central canal. Can be infratentorial (posterior fossa) or supratentorial.
Epidemiology:
- 3rd most common pediatric brain tumor.
- Peak incidence: <5 years.
Location:
- Posterior fossa (common in children)
- Ventricles or spinal canal
Clinical Features:
- Hydrocephalus → headache, nausea, vomiting
- Cranial nerve deficits if posterior fossa
Treatment & Pharmacist Role:
- Surgery – maximal safe resection is critical.
- Radiotherapy – post-op, especially if residual tumor.
- Chemotherapy – limited role, mainly for infants or unresectable tumors:
- Agents: vincristine, cisplatin, cyclophosphamide, etoposide.
- Monitor for renal, hematologic, and neurologic toxicities.
Pharmacist Notes:
- Chemotherapy often adjunctive, especially in very young children (<3y) where radiotherapy is delayed.
- Supportive care: hydration, antiemetics, growth factors as needed.
Summary Table – Key Points
| Tumor | Malignancy | Common Age | Location | Treatment Backbone | Pharmacist Pearls |
|---|---|---|---|---|---|
| Medulloblastoma | High-grade | 3–8 y | Posterior fossa | Surgery + craniospinal RT + chemo (cisplatin, vincristine, cyclophosphamide) | Monitor nephrotoxicity, neuropathy, myelosuppression; antiemetics; long-term neurocog & hearing |
| Astrocytoma | Low- or high-grade | 5–10 y | Cerebellum (low), supratentorial (high) | Surgery ± RT ± chemo (carboplatin, vincristine, temozolomide) | Supportive care focus for low-grade; monitor myelosuppression & neuropathy in chemo |
| Ependymoma | Variable | <5 y | Posterior fossa / ventricles | Surgery ± RT ± chemo (vincristine, cisplatin, cyclophosphamide, etoposide) | Chemotherapy adjunctive; toxicity monitoring; RT often delayed in infants |
Key Pharmacist Takeaways:
- Toxicity monitoring (hematologic, renal, neuro, hearing) is essential.
- Supportive care planning differs by tumor type and chemo intensity.
- Surgery and RT often the mainstay; chemo adjunctive in ependymoma, backbone in medulloblastoma/high-grade astrocytoma.
- Pediatric dosing and hydration are critical for nephrotoxic agents.
| Feature | Medulloblastoma | Astrocytoma | Ependymoma |
|---|---|---|---|
| Malignancy | High-grade | Low-/High-grade | Variable |
| Age | 3–8 y | 5–10 y | <5 y |
| Location | Posterior fossa | Cerebellum / supratentorial | Posterior fossa / ventricles / spinal |
| Surgery | Maximal safe resection | Often curative (low-grade) | Maximal safe resection |
| Radiotherapy | Craniospinal RT | High-grade or residual | Post-op, residual tumor |
| Chemo | Cisplatin, vincristine, cyclophosphamide | Carboplatin, vincristine, temozolomide | Vincristine, cisplatin, cyclophosphamide, etoposide |
| Pharmacist focus | Nephrotoxicity, neuropathy, myelosuppression | Supportive care, toxicity monitoring | Adjunct chemo, toxicity monitoring, hydration |
High-Yield Pharmacist Tips for BPS:
- Medulloblastoma → chemo backbone critical, watch kidney/nerve/bone marrow.
- Astrocytoma → low-grade may need minimal chemo, high-grade intensive therapy.
- Ependymoma → chemo adjunctive, mainly supportive care + RT timing in infants.
- Always monitor for long-term neurocognitive, endocrine, and hearing effects.
Pediatric Solid Tumors
| Tumor | Age / Epidemiology | Key Features | Treatment Backbone | Pharmacist Pearls |
|---|---|---|---|---|
| Retinoblastoma | <5 y; unilateral (sporadic) or bilateral (hereditary) | Leukocoria, strabismus | Enucleation (large), chemo-reduction: vincristine, carboplatin ± etoposide; intra-arterial/intravitreal chemo | Monitor myelosuppression, neuropathy (vincristine), renal/hearing (cisplatin). Genetic RB1 mutation → risk for secondary malignancies. |
| Neuroblastoma | <5 y; most common extracranial solid tumor | Abdominal mass, catecholamine metabolites ↑ (HVA/VMA) | Surgery, chemo (cyclophosphamide, doxorubicin, vincristine, cisplatin, etoposide), immunotherapy (anti-GD2) | Monitor myelosuppression, cardiotoxicity, nephrotoxicity. TLS prophylaxis (high-risk) with rasburicase. |
| Wilms Tumor | 2–5 y; nephroblastoma | Painless abdominal mass | Surgery + chemo: vincristine, actinomycin D ± doxorubicin; ± RT for Stage III/IV | Monitor myelosuppression, hepatotoxicity. Pre-op chemo in some protocols. Hydration with ifosfamide/cyclophosphamide. |
| Osteosarcoma | Adolescents, peak 14–18 y | Bone pain, metaphyseal tumor | Surgery + chemo: high-dose methotrexate, doxorubicin, cisplatin (MAP); ± ifosfamide/etoposide | Nephrotoxicity (cisplatin), cardiotoxicity (doxorubicin), mucositis (HD-MTX). Monitor labs, hydration, supportive care. |
| Rhabdomyosarcoma | 0–10 y | Soft tissue mass | Surgery + chemo: vincristine, actinomycin D, cyclophosphamide (VAC); ± RT | Cyclophosphamide → hemorrhagic cystitis (MESNA + hydration). Vincristine → neuropathy. Monitor blood counts. |
| Ewing Sarcoma | Adolescents | Bone/soft tissue mass | Chemo: vincristine, doxorubicin, cyclophosphamide (VDC) ± ifosfamide/etoposide (IE), surgery/RT | Monitor myelosuppression, cardiotoxicity, nephrotoxicity. Hydration and antiemetics essential. |
| Hepatoblastoma | <3 y | Abdominal mass, ↑AFP | Surgery + chemo: cisplatin ± doxorubicin | Monitor nephrotoxicity (cisplatin), cardiotoxicity (doxorubicin). Dose adjustments for infants. |
Common Pediatric Sarcomas
| Tumor | Origin | Typical Age | Key Drugs / Regimen | Pharmacist Pearls |
|---|---|---|---|---|
| Rhabdomyosarcoma (RMS) | Skeletal muscle | 0–10 y | VAC → Vincristine, Actinomycin D, Cyclophosphamide | Monitor neuropathy, myelosuppression, hemorrhagic cystitis (MESNA) |
| Osteosarcoma | Bone (metaphysis) | 10–18 y | MAP → Methotrexate (HD), Adriamycin/Doxorubicin, Cisplatin | Monitor cardiotoxicity, nephrotoxicity, mucositis, hydration |
| Ewing Sarcoma | Bone / soft tissue | 10–20 y | VDC/IE → Vincristine, Doxorubicin, Cyclophosphamide ± Ifosfamide, Etoposide | Monitor myelosuppression, cardiotoxicity, nephrotoxicity, hydration |
| Other Soft Tissue Sarcomas | Variable | Children & adolescents | Chemo depends on histology (vincristine, doxorubicin, cyclophosphamide, ifosfamide, etoposide) | Dose adjustments for age, renal/hepatic function; supportive care essential |
Willms Tumor
Definition:
- Wilms tumor (nephroblastoma) is a malignant renal tumor arising from primitive kidney (metanephric) blastemal cells.
- Most common primary kidney cancer in children.
Epidemiology
- Peak incidence: 2–5 years
- Slight female predominance
- Usually unilateral, but ~5–10% are bilateral
- Associated with syndromes: WAGR, Beckwith-Wiedemann, Denys-Drash
Clinical Features
- Abdominal mass (painless, palpable)
- Hematuria (sometimes)
- Abdominal pain, fever, hypertension
- Metastases: lungs most common
Diagnosis
- Imaging: Ultrasound, CT, or MRI of abdomen/pelvis
- Labs: Renal function, urinalysis, CBC
- Histology: blastemal, epithelial, stromal components
- Staging: NWTS/COG or SIOP protocols
Treatment & Pharmacist Role
1. Surgery
- Radical nephrectomy (unilateral)
- Nephron-sparing surgery for bilateral disease
2. Chemotherapy
- Standard-risk (unilateral, favorable histology):
- High-risk / advanced / anaplastic histology:
- Add Doxorubicin
3. Radiotherapy
- For stage III / residual disease / pulmonary metastases
4. Pharmacist Considerations
- Vincristine: neuropathy, constipation, dose-limit 2 mg/dose
- Actinomycin D: myelosuppression, hepatotoxicity
- Doxorubicin: cardiotoxicity, cumulative dose monitoring
- Supportive care: antiemetics, hydration, growth factor support if needed
- Pediatric dosing: weight- or BSA-based
High-Yield Pharmacist Pearls
- Tumor rupture before/during surgery increases stage → may require RT and intensified chemo
- Hypertension may occur due to tumor mass
- Follow-up: monitor renal function (esp. if contralateral kidney involved), cardiac function (if Doxorubicin used), and growth