UPSC MainsMEDICAL-SCIENCE-PAPER-I201910 Marks
Q25.

Give the laboratory findings in a case of Acute Myeloid leukemia (PML/RARA) fusion gene type.

How to Approach

This question requires a detailed understanding of the hematological and pathological findings specific to Acute Myeloid Leukemia (AML) with the PML/RARA fusion gene, commonly known as Acute Promyelocytic Leukemia (APL). The answer should systematically outline the findings in peripheral blood, bone marrow, and molecular studies. A structured approach, covering complete blood count (CBC), morphological examination, cytochemistry, and molecular genetic analysis, is crucial. Focus on the characteristic features that differentiate APL from other AML subtypes.

Model Answer

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Introduction

Acute Myeloid Leukemia (AML) is a heterogeneous group of malignant neoplasms of the blood and bone marrow characterized by the rapid proliferation of abnormal myeloid precursors. A specific subtype, Acute Promyelocytic Leukemia (APL), constitutes approximately 10-15% of all AML cases and is uniquely defined by the presence of the PML/RARA fusion gene resulting from a reciprocal translocation t(15;17)(q24.1;q21.2). This fusion gene disrupts normal myeloid differentiation, leading to an accumulation of promyelocytes. Accurate laboratory diagnosis is critical for prompt initiation of targeted therapy, significantly improving patient outcomes.

Laboratory Findings in Acute Myeloid Leukemia (PML/RARA) Fusion Gene Type (APL)

1. Peripheral Blood Examination

The peripheral blood findings in APL are often dramatic and characteristic:

  • White Blood Cell (WBC) Count: Typically elevated, ranging from 10,000 to >100,000/µL. However, some cases can present with normal or even decreased WBC counts.
  • Differential Count: Predominantly composed of promyelocytes (typically >90% of non-erythroid cells). Auer rods, needle-like crystalline inclusions within the cytoplasm of promyelocytes, are a hallmark finding.
  • Platelet Count: Frequently decreased (<20,000/µL) due to consumption and bone marrow suppression. Thrombocytopenia is a major contributor to bleeding complications.
  • Hemoglobin: Variable, ranging from normal to decreased, reflecting the degree of bone marrow involvement.
  • Peripheral Smear: Presence of numerous promyelocytes with characteristic Auer rods. May also observe blasts and occasional myelocytes.

2. Bone Marrow Examination

Bone marrow aspiration and biopsy are essential for confirming the diagnosis:

  • Cellularity: Typically hypercellular, with a marked proliferation of promyelocytes.
  • M:E Ratio: Significantly increased myeloid to erythroid (M:E) ratio, often >10:1.
  • Morphology: Predominance of atypical promyelocytes with abundant cytoplasm, finely granular chromatin, and prominent nucleoli. Auer rods are commonly observed.
  • Immunophenotyping: APL cells typically express myeloid markers such as CD34, CD117, and HLA-DR, but lack expression of lineage-specific markers. CD38 is often strongly positive.

3. Cytochemical Staining

Cytochemical stains can aid in the diagnosis, although less frequently used with the advent of molecular techniques:

  • Sudan Black B: Positive staining for Auer rods within the cytoplasm of promyelocytes.
  • Myeloperoxidase (MPO): Positive staining in the cytoplasm of blasts and promyelocytes, confirming myeloid lineage.
  • Periodic Acid-Schiff (PAS): Typically negative or weakly positive.

4. Molecular Genetic Studies

Molecular confirmation is crucial for definitive diagnosis and risk stratification:

  • Reverse Transcription Polymerase Chain Reaction (RT-PCR): Detects the PML/RARA fusion transcript. This is the gold standard for diagnosis.
  • Fluorescence In Situ Hybridization (FISH): Demonstrates the t(15;17) translocation directly by visualizing the fusion of the PML and RARA genes.
  • Quantitative RT-PCR (qRT-PCR): Used for monitoring minimal residual disease (MRD) during and after treatment. Higher levels of PML/RARA transcript at diagnosis and during follow-up are associated with a higher risk of relapse.

5. Coagulation Studies

APL is frequently associated with Disseminated Intravascular Coagulation (DIC):

  • Prothrombin Time (PT): Prolonged
  • Activated Partial Thromboplastin Time (aPTT): Prolonged
  • Fibrinogen: Decreased
  • D-dimer: Elevated
Test Typical Finding in APL
WBC Count Elevated (10,000 - >100,000/µL)
Promyelocytes (%) >90%
Platelet Count Decreased (<20,000/µL)
Auer Rods Present
PML/RARA Transcript (RT-PCR) Positive
PT/aPTT Prolonged

Conclusion

The laboratory findings in APL are distinctive and, when combined with clinical presentation, strongly suggest the diagnosis. Confirmation via molecular genetic studies (RT-PCR and/or FISH) is essential. Prompt diagnosis and initiation of targeted therapy, such as all-trans retinoic acid (ATRA) and arsenic trioxide (ATO), are crucial for achieving high remission rates and improving long-term survival. Monitoring for complications like DIC and MRD is also vital for optimal patient management.

Answer Length

This is a comprehensive model answer for learning purposes and may exceed the word limit. In the exam, always adhere to the prescribed word count.

Additional Resources

Key Definitions

Auer Rods
Auer rods are abnormal, crystalline inclusions found in the cytoplasm of myeloblasts and promyelocytes, particularly characteristic of acute myeloid leukemia, especially APL. They represent aggregates of abnormal primary granules.
Disseminated Intravascular Coagulation (DIC)
DIC is a life-threatening condition characterized by widespread activation of the coagulation cascade, leading to the formation of microthrombi throughout the body and subsequent consumption of clotting factors and platelets, resulting in bleeding.

Key Statistics

APL accounts for approximately 8-10% of all AML cases globally.

Source: World Health Organization Classification of Tumours of Haematopoietic and Lymphoid Tissues (2016)

Without treatment, APL is rapidly fatal, with a median survival of only weeks.

Source: American Cancer Society (Knowledge cutoff: 2023)

Examples

ATRA and APL Treatment

All-trans retinoic acid (ATRA) induces differentiation of APL blasts into mature granulocytes, effectively reversing the block in myeloid differentiation caused by the PML/RARA fusion protein. This has revolutionized APL treatment, leading to remission rates exceeding 90% when combined with arsenic trioxide.

Frequently Asked Questions

What is the significance of minimal residual disease (MRD) monitoring in APL?

MRD monitoring, typically using qRT-PCR to quantify PML/RARA transcript levels, helps assess the effectiveness of treatment and identify patients at higher risk of relapse. Persistent or increasing MRD levels may necessitate intensified therapy.

Topics Covered

PathologyHematologyLeukemiaAMLHematological Malignancies