Gemtuzumab Ozogamicin for Pediatric AML

Acute myeloid leukemia (AML) is a type of blood cancer that mostly affects older people, but in rare cases, it also affects children. Researchers are continually working to improve treatment options, and recent studies have expanded the uses of gemtuzumab ozogamicin (Mylotarg), a targeted therapy, to regimens for children with this condition.

The MyeChild 01 Trial

The MyeChild 01 trial included 515 pediatric patients, the vast majority with newly diagnosed acute myeloid leukemia. During their first course of chemotherapy, patients received either one or three doses of gemtuzumab ozogamicin. Additional chemotherapy was administered in high-risk cases. The study aimed to determine whether multiple doses could improve survival without increasing side effects.

The trial found that 94% of patients achieved complete remission after their first or second course of chemotherapy. Only 6% of patients did not respond to treatment. The two-year overall survival rate for patients in the study was 88%. 

The risk of death for those who achieved a first complete remission was lower, but 25% still experienced a relapse within two years. When comparing 1 vs 3 doses of Gemtuzumab, those who received 3 doses are estimated to stay in remission and alive (event-free survival and overall survival) significantly longer than those who received only 1 dose.

What Are the Side Effects of Gemtuzumab Ozogamicin?

The safety profile of gemtuzumab ozogamicin was closely monitored. Nearly 60% of patients experienced at least one serious side effect, consistent with expected chemotherapy-related complications. Nine patients developed severely elevated bilirubin levels, and two experienced blood clots in the liver veins (veno-occlusive disease, or VOD). This finding is important because VOD is a common side effect with the standard single dose. Notably, increasing the dosage to three doses did not lead to a higher rate or worsening of side effects..

What This Means for Patients and Families

Researching new applications of gemtuzumab ozogamicin in pediatric acute myeloid leukemia (AML) has shown encouraging results in terms of remission rates and survival. The standard use is especially convenient as it is administered as a single dose. Moving forward, it is important to continue refining the treatment strategy, focusing on dosage, drug combinations, and side effect management, to further improve outcomes in AML alongside standard chemotherapy.

Understanding the Therapies Helps Make Better Choices

Discussing all available treatment options with your healthcare team is essential to improving self-advocacy. They can help you choose suitable options based on individual health factors and disease characteristics.

Clinical trials continue to refine and improve treatment strategies, opening new possibilities for pediatric patients facing this challenging diagnosis. If you want to learn more about treatment options and clinical trials further, you can create an account in HealthTree and start exploring all our free resources. 

CREATE MY FREE ACCOUNT

Source:

• Favourable Outcomes in Newly Diagnosed Paediatric AML with Gemtuzumab Ozogamicin and Risk-Stratified Therapy: Results from the International Phase III Myechild 01 Trial