english.prescrire.org > Spotlight > Archives : 2019 > Gemtuzumab ozogamicin (Mylotarg°) in acute myeloid leukaemia: no proven benefits

Spotlight: Archives

Every month, the subjects in Prescrire’s Spotlight.

2019 : 1 | 30 | 60

Gemtuzumab ozogamicin (Mylotarg°) in acute myeloid leukaemia: no proven benefits

According to currently available clinical evaluation data, gemtuzumab ozogamicin (Mylotarg°) does not provide tangible benefits for patients with acute myeloid leukaemia.

In patients with acute myeloid leukaemia in good general condition, free of any associated disease, gemtuzumab ozogamicin has received EU approval as a first-line treatment in addition to the reference induction treatment, the combination of daunorubicin and cytarabine.

The marketing of gemtuzumab ozogamicin has seen various U-turns. Authorised in the United States of America in 2000, the company stopped marketing it in 2010 due to excess mortality resulting from adverse effects. In 2008, the European Union refused to grant a marketing authorisation for the drug in first relapse of acute myeloid leukaemia due to its unfavourable harm-benefit balance. But in 2017 in the United States, and in 2018 in the European Union, the reverse decision was taken and a marketing authorisation was granted for gemtuzumab ozogamicin as a first-line treatment in addition to the combination of daunorubicin and cytarabine, at a different dosage from that of the 2000s. These marketing authorisations were based on a trial that did not show any increase in life expectancy. A meta-analysis of five clinical trials was required to substantiate an extension of life expectancy by approximately 2 months, a result undermined by the use of variable doses of gemtuzumab ozogamicin depending on the trials.

However, gemtuzumab ozogamicin does expose patients to severe adverse effects and increased short-term mortality, including at the recently approved dosage. 

©Prescrire 1 November 2019

"Gemtuzumab ozogamicin (Mylotarg°) in acute myeloid leukaemia Uncertain effect on survival and serious adverse effects" Prescrire Int 2019; 28 (208): 263-264. (Pdf, subscribers only).

Share Share on Facebook Share on Twitter
Partager sur Facebook Partager sur Twitter

Download the full review.
Pdf, subscribers only

See also:

Midostaurin - Rydapt°.
For some types of acute
myeloid leukaemia: improved
survival, but adverse
effects underestimated
Prescrire Int 2019;
28 (201): 40.
Pdf, free

Decitabine. Acute myeloid
leukaemia: no progress
Prescrire Int 2014;
23 (148): 92-94
Pdf, subscribers only