New and Improved Leukemia Rx

Childhood leukemia responds better to new safe method of delivering medication

/ Author:  / Reviewed by: Robert Carlson, M.D

(RxWiki News) Though more common in adults, acute myeloid leukemia (AML) is the second most common type of blood cancer in children. If untreated, the disease can be fatal in just a few months.  

In AML, the bone marrow produces a large number of abnormal white blood cells that prevent healthy white blood cells from doing their job. This can lead to infection, anemia and even death.

Most patients receive an initial treatment with cancer medications such as daunorubicin (brand name Cerubidine) or idarubicin (Idamycin) followed by therapy with other cancer drugs. This combination is effective in most children but can lead to side effects on the heart muscle, among other negative outcomes, including death due to the medication.

A recent study suggests that kids with AML can be treated with higher doses of daunorubicin in a new formulation, thus improving survival rates.


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This study was conducted by Ursula Creutzig, MD, from Hannover Medical School in Hannover, Germany, and collaborators from other universities in Germany, Switzerland, Austria and Czech Republic.

The objective of the study was to see if high doses of liposomal daunorubicin would be a better alternative to standard treatment with idarubicin for patients with AML, a cancer of the blood cells.

Liposomal daunorubicin is a relatively new formulation involving injection of the medication inside liposomes, which are basically fat globules that can enclose medication molecules and enhance their absorption, thus leading to better efficacy.

"We know that the standard induction treatment regimen is effective in pediatric leukemia patients, but recognize that the toxicities associated with this therapy can be damaging to young patients who are still growing and developing," said Dr. Creutzig.

"This unique formulation of daunorubicin might offer us a way to effectively manage AML in these young patients while reducing their risk of experiencing the acute and long-term toxicities associated with traditional regimens," she said.

The researchers examined 521 AML patients less than 18 years of age between 2004 and 2010. Patients were randomly assigned to two groups, one treated with daunorbuicin and the other with idarubicin, another chemotherapy agent.

Patients in the daunorubicin group received a higher dose (80 mg/m²/day/x3) of the medication than patients treated with a medically equivalent dose of idarubicin (12 mg/m²/day/x3).

Both patient groups were also treated with the cancer medications cytarabine and etoposide. Patients who were defined as higher risk based on abnormal white blood cells and genetic abnormalities also received treatment with cytosine arabinoside, a standard cancer medication.The participants were observed over five years.

The researchers found similar results in groups treated with daunorubicin (76 percent patients survived) and idarubicin (75 percent patients survived).

The percentage of adverse events (harmful side effects) were also similar in both groups. Survival rates did not differ significantly between the normal risk groups and high risk groups.

The daunorubicin group also reported lower heart toxicities than the idarubicin treated groups. Thus, higher doses of daunorubicin had comparable safety to standard idarubicin doses and were tolerated just as well.

Treatment-related deaths were also lower in the daunorubicin treated group (2 of 257 patients) compared to the idarubicin treated group (10 of 264 patients).

"These findings signal an important step forward in our goal to identify treatments that can give pediatric patients the best chance for long-term survival with minimal toxic side effects, and we believe the approach could have a number of extended applications. For example, this treatment formulation may be appropriate to use in adults or elderly patients to reduce the toxicity profile, or it may be of value for other malignant diseases in both children and adults," said Dr. Creutzig.

"We look forward to further investigating liposomal daunorubicin as the standard anthracycline induction treatment in future studies," she said.

Dr. Manali Patel, MD, MPH from Stanford Cancer Institute, Stanford University commented on the study results, "This study is an important step in understanding how to provide treatments that can better serve our patients.  The study demonstrates efficacy of a treatment regimen and represents the first step in identifying changes in our standard protocols and regimens."

This study was published on May 23rd 2013 in Blood, the official journal of the American Society of Hematology.

The study was funded by the German Cancer Aid (Deutsche Krebshilfe) and partly by the Ministry of Health, Czech Republic. One of the authors, Dirk Reinhardt, is a member of the advisory board from Galen, a pharmaceutical company that makes liposomal daunorubicin. No other conflicts of interest or financial relationships were disclosed.

Reviewed by: 
Review Date: 
May 23, 2013
Last Updated:
August 22, 2013