Criteria

The 5th edition of the World Health Organization (WHO) classification of hematolymphoid tumors: myeloid and histiocytic/dendritic neoplasms[79]

Major criteria:

  • Hemoglobin: >16.5 g/dL in men; >16.0 g/dL in women

    or

    Hematocrit: >49% in men; >48% in women

  • Bone marrow biopsy showing hypercellularity for age with trilineage growth (panmyelosis), including prominent erythroid, granulocytic, and megakaryocytic proliferation with pleomorphic, mature megakaryocytes (differences in size)

  • Presence of Janus kinase 2 (JAK2) V617F or JAK2 exon 12 mutation

Minor criterion:

  • Subnormal serum erythropoietin level

A diagnosis of PV requires the presence of either all three major criteria, or the first two major criteria plus the minor criterion. Major criterion 2 (bone marrow biopsy) may not be required if there is sustained absolute erythrocytosis (hemoglobin levels >18.5 g/dL and >16.5 g/dL [hematocrit 55.5% and 49.5%] in men and women, respectively) if major criterion 3 and the minor criterion are present. Initial myelofibrosis (present in up to 20% of patients) can only be detected by performing a bone marrow biopsy; this finding may predict a more rapid progression to overt myelofibrosis (post-PV myelofibrosis).

International Consensus Classification of myeloid neoplasms and acute leukemias[7]

Major criteria:

  • Hemoglobin: >16.5 g/dL in men; >16.0 g/dL in women

    or

    Hematocrit: >49% in men; >48% in women

    or

    Increased red blood cell mass (>25% above mean normal predicted value)

  • Presence of JAK2 V617F or JAK2 exon 12 mutation.

  • Bone marrow biopsy showing age-adjusted hypercellularity with trilineage proliferation (panmyelosis), including prominent erythroid, granulocytic, and increase in pleomorphic, mature megakaryocytes without atypia.

Minor criterion:

  • Subnormal serum erythropoietin level.

The diagnosis of PV requires either all 3 major criteria or the first 2 major criteria plus the minor criterion. Major criterion 3 (bone marrow biopsy) may not be required in patients with sustained absolute erythrocytosis (hemoglobin concentrations of >18.5 g/dL in men or >16.5 g/dL in women and hematocrit values of >55.5% in men or >49.5% in women) and the presence of a JAK2 V617F or JAK2 exon 12 mutation.

British Committee for Standards in Haematology modified diagnostic criteria for PV[66]

Criteria:

1) JAK2-positive PV (both criteria required for diagnosis)

  • A1: Persistently raised hematocrit >52% in men, >48% in women, or raised red cell mass (>25% above predicted)

  • A2: Presence of JAK2 mutation.

2) JAK2-negative PV (diagnosis requires presence of A1-A4 plus another A or two B criteria)

  • A1: Raised red cell mass (≥25% above predicted) or hematocrit ≥60% in men, ≥56% in women

  • A2: Absence of JAK2 mutation

  • A3: No cause of secondary erythrocytosis

  • A4: Bone marrow histology consistent with PV

  • A5: Palpable splenomegaly

  • A6: Presence of acquired genetic abnormality (excluding BCR-ABL1) in hematopoietic cells

  • B1: Thrombocytosis (platelet count >450,000/microliter [450 × 10⁹/L or 450 × 10³/microliter])

  • B2: Neutrophil leukocytosis (neutrophil count >10,000/microliter [10 × 10⁹/L or 10 × 10³/microliter] in nonsmokers; >12,500/microliter [12.5 × 10⁹/L or 12.5 × 10³/microliter] in smokers)

  • B3: Radiologic evidence of splenomegaly

  • B4: Low serum erythropoietin.

Conventional risk stratification for PV[66][71][97]​​​​​

Low risk:

  • Age <60 years, and

  • No history of thrombosis.

High risk:

  • Age ≥60 years, and/or

  • History of thrombosis.

Mutation-enhanced International Prognostic Scoring System (MIPSS) for PV[83]

The MIPSS-PV is a point-based risk-stratification tool for PV that incorporates the following prognostic factors:

  • Thrombosis history (1 point)

  • Leukocyte count ≥15,000/microliter (15 × 10⁹/L or 15 × 10³/microliter) (1 point)

  • Age >67 years (2 points)

  • Spliceosome mutations (SRSF2) (3 points).

Patients with PV are stratified into the following risk groups based on total points:

  • Low risk (0-1 points)

  • Intermediate risk (2-3 points)

  • High risk (≥4 points).

The European LeukemiaNet (ELN) and International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) response criteria for polycythemia vera[98]

​Complete response is defined as:

  • Durable (lasting at least 12 weeks) resolution of disease-related signs including palpable hepatosplenomegaly, large symptom improvement (≥10-point decrease in myeloproliferative neoplasm symptom assessment form total symptom score), and[99]

  • Durable (lasting at least 12 weeks) peripheral blood count remission, defined as hematocrit <45% without phlebotomies, platelet count ≤400,000/microliter (400 × 10⁹/L or 400 × 10³/microliter), and white blood cell count <10,000/microliter (10 × 10⁹/L or 10 × 10³/microliter), and

  • Without progressive disease, and absence of any hemorrhagic or thrombotic event, and

  • Bone marrow histologic remission, defined as the presence of age-adjusted normocellularity and disappearance of trilinear hyperplasia, and absence of grade >1 reticulin fibrosis.

Fulfillment of the first three criteria above without bone marrow histologic remission, defined as the persistence of trilineage hyperplasia, constitutes a partial remission.

Progression to post-PV myelofibrosis is defined as:[100]

  • Required criteria:

    • Documentation of a previous diagnosis of PV as defined by the WHO criteria

    • Bone marrow fibrosis grade 2-3 (on 0-3 scale) or grade 3-4 (on 0-4 scale).[101][102]​​

  • Additional criteria (two are required):

    • Anemia or sustained loss of requirement of either phlebotomy (in the absence of cytoreductive therapy) or cytoreductive treatment for erythrocytosis

    • A leukoerythroblastic peripheral blood picture

    • Increasing splenomegaly, defined as either an increase in palpable splenomegaly of ≥5 cm (distance of the tip of the spleen from the left costal margin) or the appearance of a newly palpable splenomegaly

    • Development of ≥1 of 3 constitutional symptoms: >10% weight loss in 6 months, night sweats, unexplained fever (>99.5°F [>37.5°C]).

ELN formal consensus definition of clinical resistance and intolerance to hydroxyurea in polycythemia vera[103]

  • Need for phlebotomy to keep hematocrit <45% after 3 months of at least 2 g/day of hydroxyurea, OR

  • Uncontrolled myeloproliferation (i.e., platelet count >400,000/microliter [400 × 10⁹/L or 400 × 10³/microliter] and WBC count >10,000/microliter [10 × 10⁹/L or 10 × 10³/microliter]) after 3 months of at least 2 g/day of hydroxyurea, OR

  • Failure to reduce massive splenomegaly (organ extending >10 cm below the costal margin) by more than 50% as measured by palpation, or failure to completely relieve symptoms related to splenomegaly, after 3 months of at least 2 g/day of hydroxyurea, OR

  • Absolute neutrophil count <1000/microliter [1.0 × 10⁹/L or 1.0 × 10³/microliter] or platelet count <100,000/microliter (100 × 10⁹/L or 100 × 10³/microliter) or hemoglobin <10 g/dL at the lowest dose of hydroxyurea required to achieve a complete or partial clinico-hematologic response, OR

  • Presence of leg ulcers or other unacceptable hydroxyurea-related nonhematologic toxicities, such as mucocutaneous manifestations, gastrointestinal symptoms, pneumonitis, or fever at any dose of hydroxyurea.

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