Interferon α therapy for patients with essential thrombocythemia: Final results of a Phase II study initiated in 1986

Rashid Saba, Elias Jabbour, Francis Giles, Jorge Cortes, Moshe Talpaz, Susan O'Brien, Emil J. Freireich, Guillermo Garcia-Manero, Hagop Kantarjian, Srdan Verstovsek

Research output: Contribution to journalArticle

Abstract

BACKGROUND. In 1986, a Phase II trial of recombinant interferon-α (IFN-α) was initiated as therapy for patients with essential thrombocythemia (ET). METHODS. Patients were treated with subcutaneous IFN-α at a dose of 5 × 106 units/m2 daily. In responding patients, the therapy lasted at least 3 years. RESULTS. Twenty-three patients (14 females and 9 males; median age, 41 years; age range, 20-63 years) with a median platelet count of 1350 × 109/L were treated. After a median follow-up of 174 months (14.5 years), 15 of 20 evaluable patients (75%) responded, including 14 patients who achieved a complete hematologic response (CHR) (6 of them with bone marrow remission) and 1 patient who demonstrated a partial response. The median time to response was 6 months (range, 0.5-36 months), and the median response duration was 48 months (range, 5-114 months). Seven patients who achieved a CHR and were taken off therapy after they completed 3 years of maintenance therapy sustained their response for a median of 28 months. No symptoms or signs of thrombosis or hemorrhage were observed in responding patients. Eleven of 14 patients (78%) who achieved a CHR developed a recurrence, and 2 of 5 patients with recurrences who were rechallenged with IFN-α achieved a second response. The treatment was tolerated relatively well. CONCLUSIONS. IFN-α was safe and effective therapy for patients with ET, and the ability of IFN-α to reverse disease pathology and possibly modify the clinical course of patients with ET warrants its investigation in larger, prospective trials.

Original languageEnglish (US)
Pages (from-to)2551-2557
Number of pages7
JournalCancer
Volume103
Issue number12
DOIs
StatePublished - Jun 15 2005
Externally publishedYes

Fingerprint

Essential Thrombocythemia
Interferons
Therapeutics
Recurrence
Platelet Count
Signs and Symptoms

Keywords

  • Anagrelide
  • Complete hematologic response
  • Essential thrombocythemia
  • Hydroxyurea
  • Pegylated Interferon α
  • Response duration

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

Cite this

Interferon α therapy for patients with essential thrombocythemia : Final results of a Phase II study initiated in 1986. / Saba, Rashid; Jabbour, Elias; Giles, Francis; Cortes, Jorge; Talpaz, Moshe; O'Brien, Susan; Freireich, Emil J.; Garcia-Manero, Guillermo; Kantarjian, Hagop; Verstovsek, Srdan.

In: Cancer, Vol. 103, No. 12, 15.06.2005, p. 2551-2557.

Research output: Contribution to journalArticle

Saba, R, Jabbour, E, Giles, F, Cortes, J, Talpaz, M, O'Brien, S, Freireich, EJ, Garcia-Manero, G, Kantarjian, H & Verstovsek, S 2005, 'Interferon α therapy for patients with essential thrombocythemia: Final results of a Phase II study initiated in 1986', Cancer, vol. 103, no. 12, pp. 2551-2557. https://doi.org/10.1002/cncr.21086
Saba, Rashid ; Jabbour, Elias ; Giles, Francis ; Cortes, Jorge ; Talpaz, Moshe ; O'Brien, Susan ; Freireich, Emil J. ; Garcia-Manero, Guillermo ; Kantarjian, Hagop ; Verstovsek, Srdan. / Interferon α therapy for patients with essential thrombocythemia : Final results of a Phase II study initiated in 1986. In: Cancer. 2005 ; Vol. 103, No. 12. pp. 2551-2557.
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abstract = "BACKGROUND. In 1986, a Phase II trial of recombinant interferon-α (IFN-α) was initiated as therapy for patients with essential thrombocythemia (ET). METHODS. Patients were treated with subcutaneous IFN-α at a dose of 5 × 106 units/m2 daily. In responding patients, the therapy lasted at least 3 years. RESULTS. Twenty-three patients (14 females and 9 males; median age, 41 years; age range, 20-63 years) with a median platelet count of 1350 × 109/L were treated. After a median follow-up of 174 months (14.5 years), 15 of 20 evaluable patients (75{\%}) responded, including 14 patients who achieved a complete hematologic response (CHR) (6 of them with bone marrow remission) and 1 patient who demonstrated a partial response. The median time to response was 6 months (range, 0.5-36 months), and the median response duration was 48 months (range, 5-114 months). Seven patients who achieved a CHR and were taken off therapy after they completed 3 years of maintenance therapy sustained their response for a median of 28 months. No symptoms or signs of thrombosis or hemorrhage were observed in responding patients. Eleven of 14 patients (78{\%}) who achieved a CHR developed a recurrence, and 2 of 5 patients with recurrences who were rechallenged with IFN-α achieved a second response. The treatment was tolerated relatively well. CONCLUSIONS. IFN-α was safe and effective therapy for patients with ET, and the ability of IFN-α to reverse disease pathology and possibly modify the clinical course of patients with ET warrants its investigation in larger, prospective trials.",
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T2 - Final results of a Phase II study initiated in 1986

AU - Saba, Rashid

AU - Jabbour, Elias

AU - Giles, Francis

AU - Cortes, Jorge

AU - Talpaz, Moshe

AU - O'Brien, Susan

AU - Freireich, Emil J.

AU - Garcia-Manero, Guillermo

AU - Kantarjian, Hagop

AU - Verstovsek, Srdan

PY - 2005/6/15

Y1 - 2005/6/15

N2 - BACKGROUND. In 1986, a Phase II trial of recombinant interferon-α (IFN-α) was initiated as therapy for patients with essential thrombocythemia (ET). METHODS. Patients were treated with subcutaneous IFN-α at a dose of 5 × 106 units/m2 daily. In responding patients, the therapy lasted at least 3 years. RESULTS. Twenty-three patients (14 females and 9 males; median age, 41 years; age range, 20-63 years) with a median platelet count of 1350 × 109/L were treated. After a median follow-up of 174 months (14.5 years), 15 of 20 evaluable patients (75%) responded, including 14 patients who achieved a complete hematologic response (CHR) (6 of them with bone marrow remission) and 1 patient who demonstrated a partial response. The median time to response was 6 months (range, 0.5-36 months), and the median response duration was 48 months (range, 5-114 months). Seven patients who achieved a CHR and were taken off therapy after they completed 3 years of maintenance therapy sustained their response for a median of 28 months. No symptoms or signs of thrombosis or hemorrhage were observed in responding patients. Eleven of 14 patients (78%) who achieved a CHR developed a recurrence, and 2 of 5 patients with recurrences who were rechallenged with IFN-α achieved a second response. The treatment was tolerated relatively well. CONCLUSIONS. IFN-α was safe and effective therapy for patients with ET, and the ability of IFN-α to reverse disease pathology and possibly modify the clinical course of patients with ET warrants its investigation in larger, prospective trials.

AB - BACKGROUND. In 1986, a Phase II trial of recombinant interferon-α (IFN-α) was initiated as therapy for patients with essential thrombocythemia (ET). METHODS. Patients were treated with subcutaneous IFN-α at a dose of 5 × 106 units/m2 daily. In responding patients, the therapy lasted at least 3 years. RESULTS. Twenty-three patients (14 females and 9 males; median age, 41 years; age range, 20-63 years) with a median platelet count of 1350 × 109/L were treated. After a median follow-up of 174 months (14.5 years), 15 of 20 evaluable patients (75%) responded, including 14 patients who achieved a complete hematologic response (CHR) (6 of them with bone marrow remission) and 1 patient who demonstrated a partial response. The median time to response was 6 months (range, 0.5-36 months), and the median response duration was 48 months (range, 5-114 months). Seven patients who achieved a CHR and were taken off therapy after they completed 3 years of maintenance therapy sustained their response for a median of 28 months. No symptoms or signs of thrombosis or hemorrhage were observed in responding patients. Eleven of 14 patients (78%) who achieved a CHR developed a recurrence, and 2 of 5 patients with recurrences who were rechallenged with IFN-α achieved a second response. The treatment was tolerated relatively well. CONCLUSIONS. IFN-α was safe and effective therapy for patients with ET, and the ability of IFN-α to reverse disease pathology and possibly modify the clinical course of patients with ET warrants its investigation in larger, prospective trials.

KW - Anagrelide

KW - Complete hematologic response

KW - Essential thrombocythemia

KW - Hydroxyurea

KW - Pegylated Interferon α

KW - Response duration

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