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Final Results of Prospective Treatment with Pegylated Interferon Alfa-2a for Patients with Polycythemia Vera and Essential Thrombocythemia in First and Second-Line Settings

Publication ,  Conference
Yacoub, A; Mascarenhas, J; Mesa, RA; Kosiorek, HE; Rampal, RK; Silver, RT; Salama, ME; Siwoski, O; Dueck, AC; Sandy, L; McMullin, MF; Ewing, J ...
Published in: Blood
November 13, 2019

BackgroundInterferons are recognized as active agents in the treatment of patients with high risk essential thrombocythemia (ET) or polycythemia vera (PV), both in the upfront setting as well as beyond. Several trials have shown high rates of hematologic and molecular responses with the use of interferons, however, data on direct comparison of interferon activity in patients with early disease in comparison to patients refractory or resistant to prior therapies, such as hydroxyurea (HU) are lacking. We conducted a controlled analysis of the activity of pegylated interferon alfa-2a (PEG) in two prospective parallel clinical trials conducted in these two unique patient populations.MethodsThe MPD-RC 111 (NCT01259817) was an international, multicenter, phase 2 open-label clinical trial that evaluated PEG therapy in patients with high risk PV and high-risk ET who were either refractory or intolerant (R/I) to HU by modified ELN criteria. The MPD-RC 112 trial (NCT01258856) enrolled patients with high risk ET/PV who were treatment-naïve (TN) (HU <3 months) and randomized them (1:1) to PEG or HU. All patients randomized to PEG were included in this analysis. Both protocols were conducted concurrently at the MPD-RC member institutions and utilized a similar primary endpoint of overall response rate (complete and partial response rates) by ELN Criteria at 12 months confirmed by the same blinded central review committee. Both studies utilized the same PEG starting dose of 45 mcg weekly and was titrated for response to a maximum of 180 mcg weekly. Secondary endpoints included safety information, impact on disease biomarkers, bone marrow (BM) response, and quality of life data.ResultsPatientsET: 39 TN and 65 R/I ET patients were available for this analysis. Median disease duration was 2.9 months in TN and 37.3 months in R/I patients. Baseline characteristics and demographics were similar in the two cohorts except lower baseline hemoglobin level in RI patients. (Table1A)PV: 43 TN and 50 R/I PV patients were included. Median disease duration was 2.5 months in TN and 54.8 months in R/I patients. Baseline characteristics only differed by lower frequency of phlebotomy rate in R/I patients. (Table1B) Baseline symptoms scores and quality of life were similar in TN and RI groups (Table 2)ResponseET: CR/PR/ORR at 12 months were observed in 43.1%/26.2%/69.2% in R/I ET patients and in 43.6%/25.6%/69.2% in TN ET patients (p=0.99 for ORR). (Table 3, Figure 1)PV: CR/PR/ORR at 12 months were observed in 22%/38%/60% in R/I PV patients, and in 27.9%/58.1%/86% in TN PV patients (p=0.005 for ORR). (Table 3, Figure 1)SafetyPEG was equally well tolerated throughout both treatment groups with treatment discontinuation due to adverse events occurring in 14.6% in TN patients and 13.9% in R/I patients. The mean (SD) dose of PEG was 102.7 (52.3) mcg in R/I ET patients and 128.7mcg (46.4) in R/I PV patients. For TN patients, mean dose was 85.7mcg (59.7) in ET and 93.5 mcg (44.0) in PV. Adverse events were consistent with historic reports of PEG use and the distribution of events was similar in R/I and TN patients. (Table 4)ConclusionThis intention to treat response analysis included TN and R/I ET and PV patients with balanced baseline characteristics who received prospective therapy with PEG. Patients with ET had a higher overall response rate at 12 months that was equivalent in patients who were treatment-naïve and in patients who were intolerant or refractory to HU. By contrast, patients with PV who were treatment-naïve had a higher ORR than patients those intolerant or refractory to HU. We conclude that treatment with PEG is an effective therapeutic option both treatment naïve PV and ET as well as those previously treated with HU, however PEG as a second line agent is especially effective in ET patients.

Duke Scholars

Published In

Blood

DOI

EISSN

1528-0020

ISSN

0006-4971

Publication Date

November 13, 2019

Volume

134

Issue

Supplement_1

Start / End Page

2943 / 2943

Publisher

American Society of Hematology

Related Subject Headings

  • Immunology
  • 3213 Paediatrics
  • 3201 Cardiovascular medicine and haematology
  • 3101 Biochemistry and cell biology
  • 1114 Paediatrics and Reproductive Medicine
  • 1103 Clinical Sciences
  • 1102 Cardiorespiratory Medicine and Haematology
 

Citation

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Yacoub, A., Mascarenhas, J., Mesa, R. A., Kosiorek, H. E., Rampal, R. K., Silver, R. T., … Hoffman, R. (2019). Final Results of Prospective Treatment with Pegylated Interferon Alfa-2a for Patients with Polycythemia Vera and Essential Thrombocythemia in First and Second-Line Settings. In Blood (Vol. 134, pp. 2943–2943). American Society of Hematology. https://doi.org/10.1182/blood-2019-124865
Yacoub, Abdulraheem, John Mascarenhas, Ruben A. Mesa, Heidi E. Kosiorek, Raajit K. Rampal, Richard T. Silver, Mohamed E. Salama, et al. “Final Results of Prospective Treatment with Pegylated Interferon Alfa-2a for Patients with Polycythemia Vera and Essential Thrombocythemia in First and Second-Line Settings.” In Blood, 134:2943–2943. American Society of Hematology, 2019. https://doi.org/10.1182/blood-2019-124865.
Yacoub A, Mascarenhas J, Mesa RA, Kosiorek HE, Rampal RK, Silver RT, et al. Final Results of Prospective Treatment with Pegylated Interferon Alfa-2a for Patients with Polycythemia Vera and Essential Thrombocythemia in First and Second-Line Settings. In: Blood. American Society of Hematology; 2019. p. 2943–2943.
Yacoub, Abdulraheem, et al. “Final Results of Prospective Treatment with Pegylated Interferon Alfa-2a for Patients with Polycythemia Vera and Essential Thrombocythemia in First and Second-Line Settings.” Blood, vol. 134, no. Supplement_1, American Society of Hematology, 2019, pp. 2943–2943. Crossref, doi:10.1182/blood-2019-124865.
Yacoub A, Mascarenhas J, Mesa RA, Kosiorek HE, Rampal RK, Silver RT, Salama ME, Siwoski O, Dueck AC, Sandy L, McMullin MF, Ewing J, O’Connell CL, Mead AJ, De Stefano V, Weinberg RS, Baer MR, Kessler CM, Winton EF, Vannucchi AM, Kremyanskaya M, Vadakara J, Rosti V, Hexner EO, Rondelli D, Arcasoy MO, Rambaldi A, Ritchie EK, Barbui T, Kiladjian J-J, Harrison CN, Prchal JT, Hoffman R. Final Results of Prospective Treatment with Pegylated Interferon Alfa-2a for Patients with Polycythemia Vera and Essential Thrombocythemia in First and Second-Line Settings. Blood. American Society of Hematology; 2019. p. 2943–2943.

Published In

Blood

DOI

EISSN

1528-0020

ISSN

0006-4971

Publication Date

November 13, 2019

Volume

134

Issue

Supplement_1

Start / End Page

2943 / 2943

Publisher

American Society of Hematology

Related Subject Headings

  • Immunology
  • 3213 Paediatrics
  • 3201 Cardiovascular medicine and haematology
  • 3101 Biochemistry and cell biology
  • 1114 Paediatrics and Reproductive Medicine
  • 1103 Clinical Sciences
  • 1102 Cardiorespiratory Medicine and Haematology