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acute lymphoblastic leukaemia

the commonest childhood malignancy — neoplastic proliferation of lymphoid precursor cells (lymphoblasts) causing bone marrow failure with pancytopenia, bone pain, and organomegaly

haematology & oncologyrareacute

About This Page

This is a clinician-written, evidence-based summary aligned to the 2026 MLA Content Map. It is intended for medical students and junior doctors preparing for the UKMLA. Always cross-reference with NICE guidance, local protocols, and clinical judgement.

The Bottom Line

  • Commonest cancer in children (peak age 2-5 years) — also occurs in adults (second peak >60 years)
  • Presentation: pancytopenia symptoms (anaemia, infection, bleeding) + bone pain + hepatosplenomegaly + lymphadenopathy
  • Diagnosis: blood film shows lymphoblasts; bone marrow biopsy >20% blasts is diagnostic. Immunophenotyping (B-cell or T-cell ALL) essential
  • Philadelphia chromosome (BCR-ABL1 t(9;22)) present in ~25% of adult ALL — poor prognosis but responds to tyrosine kinase inhibitors
  • Treatment: intensive multi-agent chemotherapy (induction → consolidation → maintenance). CNS-directed prophylaxis essential. Childhood ALL cure rate >90%

Overview

Acute lymphoblastic leukaemia (ALL) is a malignant clonal proliferation of lymphoid precursor cells (lymphoblasts) in the bone marrow, blood, and extramedullary sites. It is classified by immunophenotype: B-cell ALL (~85% of childhood cases) and T-cell ALL (~15%). The accumulation of lymphoblasts in the marrow suppresses normal haematopoiesis, causing pancytopenia. ALL has a particular predilection for CNS involvement — hence CNS prophylaxis is a mandatory component of treatment.

Epidemiology

ALL is the commonest childhood cancer, accounting for ~25% of all paediatric malignancies. The peak incidence is age 2-5 years. Boys are slightly more commonly affected. In the UK, approximately 650-700 cases are diagnosed annually in all ages. There is a second smaller peak in adults over 60. Risk factors include Down syndrome (20-fold increased risk), prior radiation exposure, and certain genetic conditions (ataxia telangiectasia, Bloom syndrome). Childhood ALL has dramatically improved outcomes — 5-year survival now exceeds 90% in children.

Clinical Features

Symptoms
Fatigue, pallor (anaemia from marrow failure)
Recurrent infections, persistent fever (neutropenia)
Easy bruising, petechiae, epistaxis, bleeding gums (thrombocytopenia)
Bone and joint pain (marrow infiltration) — a child refusing to walk should raise suspicion
Weight loss, night sweats, malaise
Headache, vomiting, visual disturbance (CNS involvement)
Testicular swelling (testicular infiltration — sanctuary site)
Signs
Pallor, petechiae, purpura
Hepatosplenomegaly (extramedullary disease)
Generalised lymphadenopathy
Mediastinal mass (T-cell ALL — may cause SVC obstruction)
Cranial nerve palsies, papilloedema (CNS disease)
Testicular enlargement

Investigations

First-line
FBCPancytopenia common. WCC may be low, normal, or very high (hyperleukocytosis >100 × 10⁹/L — medical emergency)
Blood filmCirculating lymphoblasts — small cells with high nuclear:cytoplasmic ratio, scant cytoplasm, fine chromatin
Bone marrow aspirate and trephineDiagnostic: ≥20% lymphoblasts. Sent for morphology, immunophenotyping, cytogenetics, and molecular analysis
Second-line
Immunophenotyping (flow cytometry)Essential to classify: B-ALL (CD19+, CD10+, CD20+) or T-ALL (CD2+, CD3+, CD7+)
Cytogenetics and FISHPhiladelphia chromosome t(9;22) BCR-ABL1 (25% adults, 3-5% children — poor prognosis). t(12;21) ETV6-RUNX1 (good prognosis in children). Hyperdiploidy (good prognosis)
LDH, urate, K+, Ca2+, PO4, creatinineBaseline tumour lysis syndrome markers — check before starting treatment
Specialist
Lumbar puncture with cytospinMandatory at diagnosis — to detect CNS involvement. Blasts in CSF = CNS disease requiring intensified CNS-directed treatment
CT chestIf T-ALL suspected — look for mediastinal/thymic mass
Minimal residual disease (MRD)Flow cytometry or PCR at defined timepoints during treatment — key prognostic indicator and guides treatment intensity
1
Emergency management
  • Hyperleukocytosis (WCC >100): leucodepletion, aggressive hydration, rasburicase for tumour lysis prophylaxis
  • Neutropenic sepsis: immediate IV antibiotics (piperacillin-tazobactam per local protocol)
  • Mediastinal mass: avoid general anaesthesia (airway compression risk), urgent steroid therapy
2
Induction chemotherapy
  • Multi-agent protocol: vincristine, dexamethasone, asparaginase, ± daunorubicin
  • Aim: achieve complete remission (CR) with MRD negativity
  • Intrathecal methotrexate for CNS prophylaxis — given from induction throughout treatment
3
Consolidation and maintenance
  • Consolidation: intensification blocks with cytarabine, methotrexate, cyclophosphamide
  • Maintenance: daily 6-mercaptopurine + weekly methotrexate for 2-3 years total treatment duration
  • Philadelphia-positive ALL: add tyrosine kinase inhibitor (imatinib or dasatinib) throughout
4
Allogeneic stem cell transplant
  • Indicated for very high-risk disease, poor MRD response, or relapse
  • Philadelphia-positive ALL in adults: often transplanted in first remission
5
Relapsed/refractory ALL
  • CAR-T cell therapy (tisagenlecleucel) — NICE-approved for B-ALL in children and young adults
  • Blinatumomab (bispecific T-cell engager)
  • Inotuzumab ozogamicin (anti-CD22 antibody-drug conjugate)

Complications

  • Tumour lysis syndrome: Massive cell lysis at treatment initiation → hyperkalaemia, hyperuricaemia, hyperphosphataemia, hypocalcaemia, AKI
  • Neutropenic sepsis: Potentially fatal — the major treatment-related cause of death
  • CNS relapse: Leukaemic infiltration of meninges — hence mandatory CNS prophylaxis
  • Late effects of treatment: Avascular necrosis (steroids), cardiomyopathy (anthracyclines), secondary malignancies, infertility, neurocognitive effects
UKMLA Exam Tips
  • 1Child aged 2-5 with pancytopenia + bone pain + hepatosplenomegaly = think ALL until proven otherwise
  • 2ALL is the COMMONEST childhood cancer. AML is more common in adults
  • 3Philadelphia chromosome t(9;22) = BCR-ABL1 = poor prognosis in ALL but responds to TKIs (imatinib)
  • 4CNS involvement is characteristic of ALL — always need LP at diagnosis and intrathecal chemo for prophylaxis
  • 5Mediastinal mass = T-cell ALL. Risk of SVC obstruction and airway compromise under GA
  • 6Cure rate in childhood ALL now exceeds 90% — one of the great success stories of haematology
  • 7Tumour lysis syndrome prophylaxis: aggressive hydration + rasburicase (converts urate to allantoin)
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Verified Sources & References

NICE NG47 — Haematological Cancers: Improving Outcomes