All content on this site is intended for healthcare professionals only. By acknowledging this message and accessing the information on this website you are confirming that you are a Healthcare Professional. If you are a patient or carer, please visit Know AML.

The AML Hub uses cookies on this website. They help us give you the best online experience. By continuing to use our website without changing your cookie settings, you agree to our use of cookies in accordance with our updated Cookie Policy

Mutation testing in AML:
What you need to know

with Charles Craddock, Ralph Hills, and Gail Roboz

Wednesday, April 23, 2025
17:30-18:30 BST

Register now

This independent educational activity is supported by Thermo Fisher Scientific. All content is developed independently by the faculty. The funder is allowed no influence on the content.

  TRANSLATE

The AML Hub website uses a third-party service provided by Google that dynamically translates web content. Translations are machine generated, so may not be an exact or complete translation, and the AML Hub cannot guarantee the accuracy of translated content. The AML Hub and its employees will not be liable for any direct, indirect, or consequential damages (even if foreseeable) resulting from use of the Google Translate feature. For further support with Google Translate, visit Google Translate Help.

Steering CommitteeAbout UsNewsletterContact
LOG IN
You're logged in! Click here any time to manage your account or log out.
LOG IN
You're logged in! Click here any time to manage your account or log out.

The AML Hub is an independent medical education platform, sponsored by Astellas, Daiichi Sankyo, Johnson & Johnson, Kura Oncology and Syndax, and has been supported through educational grants from Bristol Myers Squibb and the Hippocrate Conference Institute, an association of the Servier Group. The funders are allowed no direct influence on our content. The levels of sponsorship listed are reflective of the amount of funding given. View funders.

2020-06-19T13:51:15.000Z

FDA extends approval for gemtuzumab ozogamicin in newly diagnosed CD33-positive AML

Jun 19, 2020
Share:

Bookmark this article

On June 16, 2020, the U.S. Food and Drug Administration (FDA) extended the indication of gemtuzumab ozogamicin (GO) for newly diagnosed CD33-positive acute myeloid leukemia (AML) to include pediatric patients ≥ 1 month of age. This approval was based on data from the AAML0531 trial (NCT00372593).1

Study design1,2

  • Phase III, multicenter, randomized study of 1,063 patients with newly diagnosed AML who were between the ages of 0 and 29 years
  • Aimed to compare the efficacy of GO in combination with chemotherapy with that of chemotherapy alone
  • Patients were randomized to 5-cycle chemotherapy alone or with GO administered at 3 mg/m2 once on Day 6 in the induction 1 stage and once on Day 7 of the intensification 2 stage
  • Primary endpoint: Event-free survival (EFS), measured from the date of trial entry until induction failure, relapse, or death by any cause
  • The study started in August 2006 and completed in August 2013

Results1

  • EFS hazard ratio was 0.84 (95% CI, 0.71–0.99)
  • Estimated percentage of patients free of induction failure, relapse, or death at 5 years was 48% (95% CI, 43–52) in the chemotherapy plus GO arm vs 40% (95% CI, 36–45) in the chemotherapy alone arm
  • No demonstrable difference in overall survival between treatment arms
  • The most common ≥ Grade 3 adverse reactions occurring in at least 5% of patients treated with GO included infection, febrile neutropenia, decreased appetite, hyperglycemia, mucositis, hypoxia, hemorrhage, increased transaminase, diarrhea, nausea, and hypotension

GO was previously approved by the FDA for the treatment of adult patients with newly diagnosed CD33-positive AML. Ongoing trials AML18 and AML19 are investigating how GO should be applied in AML and whether a fractionated dosing schedule can provide benefit over a single dose.

  1. S. Food and Drug Administration. FDA approves gemtuzumab ozogamicin for CD33-positive AML in pediatric patients. https://www.fda.gov/drugs/drug-approvals-and-databases/fda-approves-gemtuzumab-ozogamicin-cd33-positive-aml-pediatric-patients. Published June 16, 2020. Accessed June 17, 2020
  2. Clinicaltrials.gov. Combination chemotherapy with or without gemtuzumab in treating young patients with newly diagnosed acute myeloid leukemia. https://clinicaltrials.gov/ct2/show/record/NCT00372593 Updated April 28, 2017. Accessed June 17, 2020.

Please indicate your level of agreement with the following statements:

The content was clear and easy to understand
The content addressed the learning objectives
The content was relevant to my practice
I will change my clinical practice as a result of this content

Your opinion matters

On average, how many patients with acute myeloid leukemia do you see in a month?
7 votes - 16 days left ...

Related articles

Newsletter

Subscribe to get the best content related to AML delivered to your inbox