Polycythemia vera: The natural history of 1213 patients followed for 20 years

T. Barbui, G. Finazzi, G. De Gaetano, R. Marchioli, G. Tognoni, C. Patrono, R. Landolfi, P. Leoni, S. Rupoli, S. Cortelazzo, O. Vestri, S. Tura, C. Finelli, F. Nocentini, G. Angeli, A. Di Pasquale, V. Kramer, R. Marfisi, M. Olivieri, L. SciulliR. Spoltore, P. Rossi Ferrini, A. Grossi, G. Longo, F. De Cataldo, L. Gargantini, F. Baudo, E. M. Pagliani, I. R. Miccolis, B. Bizzi, B. Rocca, R. Tartaglione, F. Mandelli, E. Montefusco, A. Spadea, M. Carotenuto, M. Nobile, R. Morelli, L. Resegotti, M. A. Ciocca Vasino, F. Rodeghiero, M. Ruggeri, P. Rossi-Ferrini, R. M. Marfisi, Bruce R. Leslie

Research output: Contribution to journalArticle

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Abstract

Objective: To reassess the natural history of polycythemia vera and to obtain reliable estimates of both incidence of thrombosis and survival for use in defining the sample size for therapeutic clinical trials. Study Design: Rettospecttve cohort study of pattents with polycythemia who had been followed for 20 years. Setting: 11 Italian hematology institutions. Patients: 1213 patients with polycythemia vera, which was diagnosed according to criteria established by the Polycythemia Vera Study Group and commonly used in clinical practice. Main Outcome Measures: All-cause mortality, venous and arterial thrombosis, and hematologic and nonhematologic neoplastic disease. Myocardial infarction and stroke were classified as major thrombotic events, and venous and peripheral arterial thrombosis were considered minor thrombotic events. The number of patients who died and the number of those who had major thrombotic events (combined end point) were used as a comprehensive measure of the benefit-risk ratio associated with the use of myelosuppressive agents. Results: 634 fatal and nonfatal arterial and venous thromboses were recorded in 485 patients (41%); 36% of these episodes occurred during follow-up in 230 patients (19%), and 64% occurred either at presentation or before diagnosis. Thrombotic events occurred more frequently in the 2 years preceding diagnosis, suggesting a causal relation between the latent myeloproliferative disorder and the vascular event. The incidence of thrombosis during follow-up was 3.4%/y; older patients or those with a history of thrombosis had a higher risk for thrombosis. Overall mortality was 2.9/100 patients per year; thrombotic events and hematologic or nonhematologic cancers had similar effects on mortality. Patients receiving chemotherapy died three to four times more frequently than those not receiving chemotherapy. The increased risk for cancer in patients receiving myelosuppressive agents was seen approximately 6 years after diagnosis. In addition, the combined end point, computed as the sum of the hardest available events (death, nonfatal myocardial infarction, or stroke), suggests that myelosuppressive agents have an overall unfavorable effect. Conclusions: Cytoreductton favorably affects the incidence of thrombotic events, but aggressive treatment seems to be associated with increased risk for neoplasm. These results provide a basis for reevaluating the therapeutic strategy in patients with polycythemia vera and for estimating the size of clinical trials aimed at testing new therapeutic approaches.

Original languageEnglish
Pages (from-to)656-664
Number of pages9
JournalAnnals of Internal Medicine
Volume123
Issue number9
Publication statusPublished - Nov 1 1995

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Polycythemia Vera
Natural History
Thrombosis
Venous Thrombosis
Mortality
Incidence
Stroke
Myocardial Infarction
Clinical Trials
Drug Therapy
Myeloproliferative Disorders
Neoplasms
Polycythemia
Hematology
Therapeutics
Sample Size
Blood Vessels
Cohort Studies
Odds Ratio
Outcome Assessment (Health Care)

ASJC Scopus subject areas

  • Internal Medicine

Cite this

Barbui, T., Finazzi, G., De Gaetano, G., Marchioli, R., Tognoni, G., Patrono, C., ... Leslie, B. R. (1995). Polycythemia vera: The natural history of 1213 patients followed for 20 years. Annals of Internal Medicine, 123(9), 656-664.

Polycythemia vera : The natural history of 1213 patients followed for 20 years. / Barbui, T.; Finazzi, G.; De Gaetano, G.; Marchioli, R.; Tognoni, G.; Patrono, C.; Landolfi, R.; Leoni, P.; Rupoli, S.; Cortelazzo, S.; Vestri, O.; Tura, S.; Finelli, C.; Nocentini, F.; Angeli, G.; Di Pasquale, A.; Kramer, V.; Marfisi, R.; Olivieri, M.; Sciulli, L.; Spoltore, R.; Rossi Ferrini, P.; Grossi, A.; Longo, G.; De Cataldo, F.; Gargantini, L.; Baudo, F.; Pagliani, E. M.; Miccolis, I. R.; Bizzi, B.; Rocca, B.; Tartaglione, R.; Mandelli, F.; Montefusco, E.; Spadea, A.; Carotenuto, M.; Nobile, M.; Morelli, R.; Resegotti, L.; Ciocca Vasino, M. A.; Rodeghiero, F.; Ruggeri, M.; Rossi-Ferrini, P.; Marfisi, R. M.; Leslie, Bruce R.

In: Annals of Internal Medicine, Vol. 123, No. 9, 01.11.1995, p. 656-664.

Research output: Contribution to journalArticle

Barbui, T, Finazzi, G, De Gaetano, G, Marchioli, R, Tognoni, G, Patrono, C, Landolfi, R, Leoni, P, Rupoli, S, Cortelazzo, S, Vestri, O, Tura, S, Finelli, C, Nocentini, F, Angeli, G, Di Pasquale, A, Kramer, V, Marfisi, R, Olivieri, M, Sciulli, L, Spoltore, R, Rossi Ferrini, P, Grossi, A, Longo, G, De Cataldo, F, Gargantini, L, Baudo, F, Pagliani, EM, Miccolis, IR, Bizzi, B, Rocca, B, Tartaglione, R, Mandelli, F, Montefusco, E, Spadea, A, Carotenuto, M, Nobile, M, Morelli, R, Resegotti, L, Ciocca Vasino, MA, Rodeghiero, F, Ruggeri, M, Rossi-Ferrini, P, Marfisi, RM & Leslie, BR 1995, 'Polycythemia vera: The natural history of 1213 patients followed for 20 years', Annals of Internal Medicine, vol. 123, no. 9, pp. 656-664.
Barbui T, Finazzi G, De Gaetano G, Marchioli R, Tognoni G, Patrono C et al. Polycythemia vera: The natural history of 1213 patients followed for 20 years. Annals of Internal Medicine. 1995 Nov 1;123(9):656-664.
Barbui, T. ; Finazzi, G. ; De Gaetano, G. ; Marchioli, R. ; Tognoni, G. ; Patrono, C. ; Landolfi, R. ; Leoni, P. ; Rupoli, S. ; Cortelazzo, S. ; Vestri, O. ; Tura, S. ; Finelli, C. ; Nocentini, F. ; Angeli, G. ; Di Pasquale, A. ; Kramer, V. ; Marfisi, R. ; Olivieri, M. ; Sciulli, L. ; Spoltore, R. ; Rossi Ferrini, P. ; Grossi, A. ; Longo, G. ; De Cataldo, F. ; Gargantini, L. ; Baudo, F. ; Pagliani, E. M. ; Miccolis, I. R. ; Bizzi, B. ; Rocca, B. ; Tartaglione, R. ; Mandelli, F. ; Montefusco, E. ; Spadea, A. ; Carotenuto, M. ; Nobile, M. ; Morelli, R. ; Resegotti, L. ; Ciocca Vasino, M. A. ; Rodeghiero, F. ; Ruggeri, M. ; Rossi-Ferrini, P. ; Marfisi, R. M. ; Leslie, Bruce R. / Polycythemia vera : The natural history of 1213 patients followed for 20 years. In: Annals of Internal Medicine. 1995 ; Vol. 123, No. 9. pp. 656-664.
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abstract = "Objective: To reassess the natural history of polycythemia vera and to obtain reliable estimates of both incidence of thrombosis and survival for use in defining the sample size for therapeutic clinical trials. Study Design: Rettospecttve cohort study of pattents with polycythemia who had been followed for 20 years. Setting: 11 Italian hematology institutions. Patients: 1213 patients with polycythemia vera, which was diagnosed according to criteria established by the Polycythemia Vera Study Group and commonly used in clinical practice. Main Outcome Measures: All-cause mortality, venous and arterial thrombosis, and hematologic and nonhematologic neoplastic disease. Myocardial infarction and stroke were classified as major thrombotic events, and venous and peripheral arterial thrombosis were considered minor thrombotic events. The number of patients who died and the number of those who had major thrombotic events (combined end point) were used as a comprehensive measure of the benefit-risk ratio associated with the use of myelosuppressive agents. Results: 634 fatal and nonfatal arterial and venous thromboses were recorded in 485 patients (41{\%}); 36{\%} of these episodes occurred during follow-up in 230 patients (19{\%}), and 64{\%} occurred either at presentation or before diagnosis. Thrombotic events occurred more frequently in the 2 years preceding diagnosis, suggesting a causal relation between the latent myeloproliferative disorder and the vascular event. The incidence of thrombosis during follow-up was 3.4{\%}/y; older patients or those with a history of thrombosis had a higher risk for thrombosis. Overall mortality was 2.9/100 patients per year; thrombotic events and hematologic or nonhematologic cancers had similar effects on mortality. Patients receiving chemotherapy died three to four times more frequently than those not receiving chemotherapy. The increased risk for cancer in patients receiving myelosuppressive agents was seen approximately 6 years after diagnosis. In addition, the combined end point, computed as the sum of the hardest available events (death, nonfatal myocardial infarction, or stroke), suggests that myelosuppressive agents have an overall unfavorable effect. Conclusions: Cytoreductton favorably affects the incidence of thrombotic events, but aggressive treatment seems to be associated with increased risk for neoplasm. These results provide a basis for reevaluating the therapeutic strategy in patients with polycythemia vera and for estimating the size of clinical trials aimed at testing new therapeutic approaches.",
author = "T. Barbui and G. Finazzi and {De Gaetano}, G. and R. Marchioli and G. Tognoni and C. Patrono and R. Landolfi and P. Leoni and S. Rupoli and S. Cortelazzo and O. Vestri and S. Tura and C. Finelli and F. Nocentini and G. Angeli and {Di Pasquale}, A. and V. Kramer and R. Marfisi and M. Olivieri and L. Sciulli and R. Spoltore and {Rossi Ferrini}, P. and A. Grossi and G. Longo and {De Cataldo}, F. and L. Gargantini and F. Baudo and Pagliani, {E. M.} and Miccolis, {I. R.} and B. Bizzi and B. Rocca and R. Tartaglione and F. Mandelli and E. Montefusco and A. Spadea and M. Carotenuto and M. Nobile and R. Morelli and L. Resegotti and {Ciocca Vasino}, {M. A.} and F. Rodeghiero and M. Ruggeri and P. Rossi-Ferrini and Marfisi, {R. M.} and Leslie, {Bruce R.}",
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TY - JOUR

T1 - Polycythemia vera

T2 - The natural history of 1213 patients followed for 20 years

AU - Barbui, T.

AU - Finazzi, G.

AU - De Gaetano, G.

AU - Marchioli, R.

AU - Tognoni, G.

AU - Patrono, C.

AU - Landolfi, R.

AU - Leoni, P.

AU - Rupoli, S.

AU - Cortelazzo, S.

AU - Vestri, O.

AU - Tura, S.

AU - Finelli, C.

AU - Nocentini, F.

AU - Angeli, G.

AU - Di Pasquale, A.

AU - Kramer, V.

AU - Marfisi, R.

AU - Olivieri, M.

AU - Sciulli, L.

AU - Spoltore, R.

AU - Rossi Ferrini, P.

AU - Grossi, A.

AU - Longo, G.

AU - De Cataldo, F.

AU - Gargantini, L.

AU - Baudo, F.

AU - Pagliani, E. M.

AU - Miccolis, I. R.

AU - Bizzi, B.

AU - Rocca, B.

AU - Tartaglione, R.

AU - Mandelli, F.

AU - Montefusco, E.

AU - Spadea, A.

AU - Carotenuto, M.

AU - Nobile, M.

AU - Morelli, R.

AU - Resegotti, L.

AU - Ciocca Vasino, M. A.

AU - Rodeghiero, F.

AU - Ruggeri, M.

AU - Rossi-Ferrini, P.

AU - Marfisi, R. M.

AU - Leslie, Bruce R.

PY - 1995/11/1

Y1 - 1995/11/1

N2 - Objective: To reassess the natural history of polycythemia vera and to obtain reliable estimates of both incidence of thrombosis and survival for use in defining the sample size for therapeutic clinical trials. Study Design: Rettospecttve cohort study of pattents with polycythemia who had been followed for 20 years. Setting: 11 Italian hematology institutions. Patients: 1213 patients with polycythemia vera, which was diagnosed according to criteria established by the Polycythemia Vera Study Group and commonly used in clinical practice. Main Outcome Measures: All-cause mortality, venous and arterial thrombosis, and hematologic and nonhematologic neoplastic disease. Myocardial infarction and stroke were classified as major thrombotic events, and venous and peripheral arterial thrombosis were considered minor thrombotic events. The number of patients who died and the number of those who had major thrombotic events (combined end point) were used as a comprehensive measure of the benefit-risk ratio associated with the use of myelosuppressive agents. Results: 634 fatal and nonfatal arterial and venous thromboses were recorded in 485 patients (41%); 36% of these episodes occurred during follow-up in 230 patients (19%), and 64% occurred either at presentation or before diagnosis. Thrombotic events occurred more frequently in the 2 years preceding diagnosis, suggesting a causal relation between the latent myeloproliferative disorder and the vascular event. The incidence of thrombosis during follow-up was 3.4%/y; older patients or those with a history of thrombosis had a higher risk for thrombosis. Overall mortality was 2.9/100 patients per year; thrombotic events and hematologic or nonhematologic cancers had similar effects on mortality. Patients receiving chemotherapy died three to four times more frequently than those not receiving chemotherapy. The increased risk for cancer in patients receiving myelosuppressive agents was seen approximately 6 years after diagnosis. In addition, the combined end point, computed as the sum of the hardest available events (death, nonfatal myocardial infarction, or stroke), suggests that myelosuppressive agents have an overall unfavorable effect. Conclusions: Cytoreductton favorably affects the incidence of thrombotic events, but aggressive treatment seems to be associated with increased risk for neoplasm. These results provide a basis for reevaluating the therapeutic strategy in patients with polycythemia vera and for estimating the size of clinical trials aimed at testing new therapeutic approaches.

AB - Objective: To reassess the natural history of polycythemia vera and to obtain reliable estimates of both incidence of thrombosis and survival for use in defining the sample size for therapeutic clinical trials. Study Design: Rettospecttve cohort study of pattents with polycythemia who had been followed for 20 years. Setting: 11 Italian hematology institutions. Patients: 1213 patients with polycythemia vera, which was diagnosed according to criteria established by the Polycythemia Vera Study Group and commonly used in clinical practice. Main Outcome Measures: All-cause mortality, venous and arterial thrombosis, and hematologic and nonhematologic neoplastic disease. Myocardial infarction and stroke were classified as major thrombotic events, and venous and peripheral arterial thrombosis were considered minor thrombotic events. The number of patients who died and the number of those who had major thrombotic events (combined end point) were used as a comprehensive measure of the benefit-risk ratio associated with the use of myelosuppressive agents. Results: 634 fatal and nonfatal arterial and venous thromboses were recorded in 485 patients (41%); 36% of these episodes occurred during follow-up in 230 patients (19%), and 64% occurred either at presentation or before diagnosis. Thrombotic events occurred more frequently in the 2 years preceding diagnosis, suggesting a causal relation between the latent myeloproliferative disorder and the vascular event. The incidence of thrombosis during follow-up was 3.4%/y; older patients or those with a history of thrombosis had a higher risk for thrombosis. Overall mortality was 2.9/100 patients per year; thrombotic events and hematologic or nonhematologic cancers had similar effects on mortality. Patients receiving chemotherapy died three to four times more frequently than those not receiving chemotherapy. The increased risk for cancer in patients receiving myelosuppressive agents was seen approximately 6 years after diagnosis. In addition, the combined end point, computed as the sum of the hardest available events (death, nonfatal myocardial infarction, or stroke), suggests that myelosuppressive agents have an overall unfavorable effect. Conclusions: Cytoreductton favorably affects the incidence of thrombotic events, but aggressive treatment seems to be associated with increased risk for neoplasm. These results provide a basis for reevaluating the therapeutic strategy in patients with polycythemia vera and for estimating the size of clinical trials aimed at testing new therapeutic approaches.

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