Table 2. Studies evaluating outcomes in polycythemia vera.
| Kiladjian et al.7 | Passamonti et al.11 | Finazzi et al.12 | Marchioli et al.13 | Gangat et al.14 | Passamonti et al.15 | Abdulkarim et al.16 | Tefferi et al.3 | Bonicelli et al.17 | Stein et al.18 | Sever et al.19 | Tefferi et al.2 | Bai et al.20 | |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| N | 164 | 396 | 1638 | 1638 | 459 | 320 | 150 | 1545 | 327 | 204 | 133 | 267 (Mayo) 310 (Italy) | 272 |
| Median F/U (years) | 11.4 | 9.6 | 2.8 | 2.7 | 5.3 | 3.2 | 15 | 6.9 | 11 | 8 (age ⩽45 years) 4.5 (age ⩾65 years) | 7.5 | 11.8 11.1 | 6 |
| MF | |||||||||||||
| N | 14 (8.5%) | 5.1 (3.3–7.8)a | NR | 38 (2.3%) | 54 (12%) | 8 (2.5%) | 13 (8.5%) | 138 (9%) | 37 (11%) | 26 (12.7%) | 11 (8%) | 34 (12.7%) 65 (21%) | 63 (23%)b |
| Median time to MF from Dx (years): | 12.5 | 13 | NR | NR | 10.5 | NR | NR | NR | 9.6 | 20 (age ⩽45 years) 8 (age ⩾65 years) | 8.5c | 9.6 | NR |
| Risk factors | None | Sequential use of ⩾2 myelosuppressive agents compared with Pipobroman | NR | Disease duration >10 years | Age ⩾60 years | JAK2V617F allele burden ⩾50% | Splenomegaly Reticulin grading | NR | None | Median age at MF Dx | None | None | Splenomegaly Plt>550 × 109/l JAK2V617F allele burden ⩾50% |
| Leukemia | |||||||||||||
| N | 32 (19.5%) | 5.3 (3.5-8)a | 22 (1.3%) | 21 (1.3%) | 34 (7.4%) | 10 (3%) | 13 (8.5%) | 50 (3%) | 30 (9.2%) | 7 (3%) | 4 (3%) | 18 (6.7%) 10 (3.2%) | 6.6 (3.7-10.8)d |
| Median time to LT from Dx (years): | 9.6 | 14 | 8.4 | NR | 10.5 | NR | NR | 10.8 | 4.6 | 19 (age ⩽45 years) 7 (age ⩾65 years) | 8.5c | NR | NR |
| Risk factors | WBC⩾10 × 109/l | Sequential use of ⩾2 myelosuppressive agents compared with HU or Pipobroman | Age ⩾70 years P32 Busulphan Pipobroman Cytoreductive drug alone or in combo Low cholesterol (⩽150 mg/dl) | Age ⩾70 years Cytoreductive agents other than HU or IFN | WBC⩾10 × 109/l WBC⩾15 × 109/l | None | Splenomegaly Reticulin grading | Age >61 years WBC ⩾15 × 109/l Abnormal karyotype P32/CMB alone Pipobroman | Female | None | None | None | Plt<100 × 109/l |
| Median Survival (years): | 15.5 | 20 | NR | NRe | 22.7 | NR | NRf | 18.9g | 17.5 (age <65 years) 6.4 (age ⩾65 years)h | NR | 24 | 13.5i | NRj |
| Risk factors | Age ⩾60 years WBC⩾10 × 109/l | Thrombosis | NR | Age >65 years Thrombosis | Age ⩾60 years WBC⩾15 × 109/l Arterial thrombosis | Age ⩾60 years | Splenomegaly | Age >61 years WBC⩾15 × 109/l Thrombosis Abnormal karyotype Pruritusk | Age >70 years WBC>13 × 109/l Thrombosis | NR | None | Age <60 years | Age >65 years WBC>25 × 109/l Thrombosis |
Abbreviations: CMB, chlorambucil; Dx, diagnosis; HU, hydroxyurea; IFN, interferon; LT, leukemic transformation; MF, myelofibrosis; NR, not reported; WBC, white blood cell.
a
Incidence per 1000 person-years (95% CI).
b
Incidence of 31 (95% CI=24.12–40.16) per 1000 person/year.
c
Median time to leukemic or myelofibrotic transformation.
d
Incidence rate per 1000 person/year with 19% leukemic transformation from post-PV MF population (n=63).
e
Overall mortality rate of 3.7 per 100 persons per year.
f
Median overall survival 40% at 10 years.
g
Median overall survival 14.1 years based on most mature follow-up cohort (n=337).
h
Median overall survival 72% at 10 years.
i
Based on Mayo cohort.
j
83% median overall survival at 10 years.
k
Favorable risk factor.