Efficacy and safety of anagrelide as a first‐line drug in cytoreductive treatment‐naïve essential thrombocythemia patients in a real‐world setting

Abstract

Objective: This study aimed to retrospectively assess the efficacy and safety of anagrelide in cytoreduction therapy-naïve essential thrombocythemia (ET) patients in a real-world setting.Method: Data from 53 ET patients who received anagrelide as a first-line therapy were reviewed for patient characteristics, antiplatelet status, cytoreduction status, therapeutic effects, adverse events, thrombohemorrhagic event development, progression to myelofibrosis or acute leukemia, and cause of death. Results:The rate of achieving a platelet count of <600 × 10 9 /L during anagrelide monotherapy was 83.0%. Adverse events occurred in 32 of 53 patients, and tended to be slightly more severe in patients with cardiac failure; however, they were mostly tolerable. The therapeutic effect of anagrelide was consistent, regardless of genetic mutation profiles. The incidence of anemia as an adverse event was significantly higher in the CALR mutation-positive group. Favorable platelet counts were also achieved in patients for whom hydroxyurea was introduced as a replacement for anagrelide or in addition to anagrelide because of unresponsiveness or intolerance to treatment. Conclusion:In Japanese cytoreduction therapy-naïve ET patients, anagrelide administration as a first-line therapy demonstrated favorable effects in reducing platelet counts, and its safety profile that was generally consistent with those in previous reports. K E Y W O R D S anagrelide, essential thrombocythemia, first-line, treatment | 117 ITO eT al. Complete response, n (%) 27 (50.9) Partial response, n (%) 18 (34.0) No response, n (%) 8 (15.1) AEs (≥5% of patients) Palpitations, n (%) 14 (26.4) Headache, n (%) 11 (20.8) Anemia, n (%) 10 (18.9) Diarrhea, n (%) 4 (7.5) Cardiac failure, n (%) 3 (5.7) AEs (grade 3) Anemia, n (%) 2 (3.8) Cardiac failure, n (%) 2 (3.8) Abbreviations: AE: adverse event; SD: standard deviation.

Interferon alpha therapy in essential thrombocythemia and polycythemia vera—a systematic review and meta-analysis

“…This is especially relevant as alternative therapies such as hydroxyurea, phlebotomy, or anagrelide seem to have less side effects and are effective in reducing the risk of thromboembolic complications. 4 , 5 , 67 – 70 As there are currently no large randomized trial data showing superiority of IFN over either anagrelide or hydroxyurea available, both the National Comprehensive Cancer Network (NCCN) and European Leukemia Net (ELN) recommend hydroxyurea over IFN for cytoreduction in ET patients. 1 , 63 However, recommendations differ for PV patients with NCCN recommending hydroxyurea in addition to ASA and phlebotomy for high-risk patients (age ≥60 years and/or prior thrombosis) with IFN as an alternative mainly for younger or pregnant patients.…”

“Anagrelide‐induced pulmonary arterial hypertension”: a rare case of drug‐induced pulmonary arterial hypertension

“…4,5 Anagrelide (Agrylin Õ ) is an oral imidazoquinazoline agent that is prescribed for reducing elevated platelet counts and ameliorating thrombohemorrhagic events in patients with myeloproliferative disorders. 6 Here, we report the case of a 70-year-old female patient who developed PAH associated with anagrelide use for the treatment of polycythemia vera.…”

“…Anagrelide (Agrylin®) is an oral imidazoquinazoline agent that is prescribed for reducing elevated platelet counts and ameliorating thrombohemorrhagic events in patients with myeloproliferative disorders 6 …”

Safety and efficacy of anagrelide in Japanese post-marketing surveillance, with subgroup analyses on the effect of previous cytoreductive therapies, age, and starting dose

“…Anagrelide has also been approved for treatment-naïve patients with ET based on the results of two global clinical studies; ANAHYDRET and a Phase IIIb clinical study for anagrelide [ 13 , 14 ]. Therefore, the safety and efficacy findings for anagrelide in CRT-naïve Japanese patients are limited [ 15 , 16 ].…”