Main epidemiological studies in polycythemia vera (PV).
| Study . | Design, No. of Patients, Follow-up . | Treatments . | Results, Comments . |
|---|---|---|---|
| Abbreviations: ET, essential thrombocythemia, HU, hydroxyurea, AML, acute myeloid leukemia; O/E, observed events to the events expected in an age- and sex-matched reference population | |||
| Brussamolino1 | Cohort prospective study on 100 PV pts. Median follow-up 5 years | Pipobroman alone or in association | Median survival 140 months. Death rate at 12 years: 23%. Risk of AML at 5 years: 6%. Risk of AML at 7 years: 9%. |
| Najean2,3 | Cohort prospective study on 175 PV pts. Follow-up 10 years. | Reduced dose of 32P ± low-dose HU | Survival at 5 years: 32P alone: 88%; 32P + HU: 79% Survival at 7 years: 32P alone: 77%; 32P + HU: 66% |
| Lofvenberg4 | Cohort prospective study on 59 pts with PV, ET, myelofibrosis | HU | After 5 years survival (> 86%) similar to a sex- and age-matched Swedish population. Thrombotic event incidence: 20.8%. |
| Weinfeld5 | 38 pts with PV, ET. Median follow-up > 10 years | HU | AML in 4/38 (10.5%) |
| Lofvenberg6 | 81 PV and ET pts. Median follow-up 4 years | HU | AML in 4/65 (6.2%) |
| Najean7 | 96 PV pts. Median follow-up 5.3 years. Pipobroman | HU | Absence of carcinogenic risk at short- but not necessarily at long-term |
| Messinezy8 | Retrospective study on 65 PV pts. 20 years follow-up | Venesection + low-dose busulfan | Median survival 11.1 years. O/E = 1.86 (P < .05). AML incidence 3.5%. |
| Rozman9 | Retrospective study on 1067 pts (PV 43%; ET 23%; M 34%). Median follow-up 4.3 years. | — | Observed survival not different from an age- and sex-matched Spanish population for PV and ET, but significantly worse for myelofibrosis. O/E in PV: 1.01; O/E in ET: 1.22; O/E in myelofibrosis: 1.59; |
| Brandt10 | Retrospective on 366 PV or ET pts. Median follow-up 7.2 years | 32P#32P | 162/366 deaths. O/E = .38. 17/366 cases of AML (4.6%), apparent accumulation of AML cases 8–12 years after start of treatment. Excess mortality 8–10 years after start of treatment |
| GISP11 | Retrospective study on 1213 PV pts. Median follow-up 5.3 years | — | Death rate 2.94 per 100 PY. O/E = 1.69 (P < .05). Increase in risk of malignancies 6 years after diagnosis in pts receiving chemo-therapy agents. Adjusted RR of death and major non-fatal thrombosis 2.1 (95%CI 1.4–3.1) |
| Study . | Design, No. of Patients, Follow-up . | Treatments . | Results, Comments . |
|---|---|---|---|
| Abbreviations: ET, essential thrombocythemia, HU, hydroxyurea, AML, acute myeloid leukemia; O/E, observed events to the events expected in an age- and sex-matched reference population | |||
| Brussamolino1 | Cohort prospective study on 100 PV pts. Median follow-up 5 years | Pipobroman alone or in association | Median survival 140 months. Death rate at 12 years: 23%. Risk of AML at 5 years: 6%. Risk of AML at 7 years: 9%. |
| Najean2,3 | Cohort prospective study on 175 PV pts. Follow-up 10 years. | Reduced dose of 32P ± low-dose HU | Survival at 5 years: 32P alone: 88%; 32P + HU: 79% Survival at 7 years: 32P alone: 77%; 32P + HU: 66% |
| Lofvenberg4 | Cohort prospective study on 59 pts with PV, ET, myelofibrosis | HU | After 5 years survival (> 86%) similar to a sex- and age-matched Swedish population. Thrombotic event incidence: 20.8%. |
| Weinfeld5 | 38 pts with PV, ET. Median follow-up > 10 years | HU | AML in 4/38 (10.5%) |
| Lofvenberg6 | 81 PV and ET pts. Median follow-up 4 years | HU | AML in 4/65 (6.2%) |
| Najean7 | 96 PV pts. Median follow-up 5.3 years. Pipobroman | HU | Absence of carcinogenic risk at short- but not necessarily at long-term |
| Messinezy8 | Retrospective study on 65 PV pts. 20 years follow-up | Venesection + low-dose busulfan | Median survival 11.1 years. O/E = 1.86 (P < .05). AML incidence 3.5%. |
| Rozman9 | Retrospective study on 1067 pts (PV 43%; ET 23%; M 34%). Median follow-up 4.3 years. | — | Observed survival not different from an age- and sex-matched Spanish population for PV and ET, but significantly worse for myelofibrosis. O/E in PV: 1.01; O/E in ET: 1.22; O/E in myelofibrosis: 1.59; |
| Brandt10 | Retrospective on 366 PV or ET pts. Median follow-up 7.2 years | 32P#32P | 162/366 deaths. O/E = .38. 17/366 cases of AML (4.6%), apparent accumulation of AML cases 8–12 years after start of treatment. Excess mortality 8–10 years after start of treatment |
| GISP11 | Retrospective study on 1213 PV pts. Median follow-up 5.3 years | — | Death rate 2.94 per 100 PY. O/E = 1.69 (P < .05). Increase in risk of malignancies 6 years after diagnosis in pts receiving chemo-therapy agents. Adjusted RR of death and major non-fatal thrombosis 2.1 (95%CI 1.4–3.1) |