Efficacy

The Phase III ALFA-0701 study was an open-label, registrational study evaluating MYLOTARG in combination with standard front-line chemotherapy versus standard front-line chemotherapy for patients with de novo acute myeloid leukaemia (AML).^1,2

The intention-to-treat (ITT) population included 280 treatment-naive patients*, aged 50-70 years old, with confirmed AML and normal cardiac function^† that were randomised 1:1 to receive either MYLOTARG 3mg/m² (maximum one 5mg vial) on days 1, 4 and 7 plus daunorubicin/cytarabine (DNR/AraC) 3+7 (n=135*) OR DNR/AraC 3+7 alone (n=136*).^1-4
 

The primary endpoint was event‑free survival (EFS), defined as the time from randomisation to relapse, death from any cause, or time to the date of assessment of response if complete remission (CR) or complete remission with incomplete platelet recovery (CRp) had not been achieved.²
 

^*All 280 patients were randomised (ITT); 9 were excluded post‑randomisation due to inadequate consent documentation, resulting in a modified ITT population of 271 patients. Data are from the extended follow-up analysis (cut-off: 30 April 2013) reporting final overall survival results from ALFA-0701; full patient flow is reported in Lambert J, et al. Haematologica 2019.<sup>3,4

†</sup>Three randomised patients did not receive assigned treatment (MYLOTARG + DNR/AraC, n=1; DNR/AraC alone, n=2) due to death or eligibility violations (esophageal cancer or hepatitis B).

^‡Patients did not receive MYLOTARG during first induction due to abnormal liver function, failure to meet eligibility criteria, or death.

^§Eligible patients required a locally confirmed morphological diagnosis of AML and normal cardiac function, assessed by use of radionucleotide scintigraphy or echography. CD33+ leukaemic blast cells were not required for study entry.<sup>2

¶</sup>Original study allowed consolidation for patients with a complete remission with incomplete platelet recovery (CRp) but dosing modifications preclude the use of MYLOTARG for consolidation in patients with a platelet count of <100,000/mm³.<sup>1,2

‖</sup>Without MYLOTARG and in patients who did not achieve CR/CRp after Course 1.^2,3

Median EFS was significantly longer with MYLOTARG^® + standard chemotherapy vs standard chemotherapy alone³

A retrospective analysis of the 235 patients with non‑CBF AML from the ALFA‑0701 study assessed the efficacy of MYLOTARG plus standard front-line chemotherapy according to ELN 2017 risk classification.^5,6

Results showed that the addition of MYLOTARG to standard front-line chemotherapy prolonged EFS vs standard front-line chemotherapy alone in patients who had favourable‑risk and intermediate‑risk cytogenetics, with no benefit shown in those with adverse cytogenetics.⁷

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Abbreviations:
AML, acute myeloid leukaemia; AraC, cytarabine; CD33, cluster of differentiation 33; CI, confidence interval; CR, complete remission; CRp, complete remission with incomplete platelet recovery; DGH, District General Hospital; DNR, daunorubicin; EFS, event‑free survival; ELN, European LeukemiaNet; HR, hazard ratio; IC, intensive chemotherapy; ITT, intention‑to‑treat; mEFS, median event-free survival; mRFS, median relapse-free survival; OS, overall survival. 


References:

1. MYLOTARG Summary of Product Characteristics for the United Kingdom click here.

2. Castaigne S, et al. Lancet 2012;379:1508–1516

3. Lambert J, et al. Haematologica 2019;104:113–119

4. Lambert J, et al. Haematologica 2019;104:113–119. Supplementary analysis

5. Fournier E, et al. Blood 2020;135(8):542–546

6. Döhner H, et al. Blood 2022; 140(12): 1345–1377.

7. Pfizer Ltd. Data on file. REF-MYL1183