Health # FDA approves liposomal daunorubicin-cytarabine combination for Blood Cancer(Poor Prognosis type of AML)

Hello my steemit friends.Welcome to my new post in the health series.

Earlier this week FDA(Food and Drug Administration) has approved the use of liposomal daunorubicin-cytarabine combination for the Blood Cancer therapy specifically speaking ,the AML(Acute myloid lukaemia).

Daunorubicin-Cytarabine combination therapy is the widely used regimen for the treatment of different types of AML, but this new approval is specifically targeted for the Therapy induced AML and Myelodysplastic AML.

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The official FDA statement

On August 3, 2017, the U.S. Food and Drug Administration granted regular approval to a liposome-encapsulated combination of daunorubicin and cytarabine (VYXEOS, Jazz Pharmaceuticals, Inc.) for the treatment of adults with newly-diagnosed therapy-related AML (t-AML) or AML with myelodysplasia-related changes (AML-MRC), two types of AML having a poor prognosis.
This is the first FDA-approved treatment specifically for patients with t-AML or AML-MRC.

Approval was based on data from Study CLTR0310-301 (NCT01696084), a randomized (1:1), multicenter, open-label, active-controlled trial comparing Vyxeos to a standard combination of daunorubicin and cytarabine (7+3) in 309 patients 60-75 years of age with newly-diagnosed t-AML or AML-MRC. Vyxeos demonstrated an estimated median overall survival of 9.6 months compared with 5.9 months for the 7+3 control (hazard ratio 0.69; 95% CI: 0.52, 0.90; p=0.005).[1]

What is AML? How Bad is it?

AML(Acute Myloid lukaemia) is a serious type of blood cancer which affects the blood forming cells in the bone marrow. Bone marrow is a substance present inside the bone which contains stem cells responsible for formation of various types of blood cells like- Red Blood Cells,White blood cells and the Platelets.

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The picture above shows the normal development of Blood Cells, the stem cells are present in the bone marrow, they differentiate into different types of Blood cells as shown in the figure.

When mutation occurs in these stem cells, they become malignant i.e. they proliferate excessively and abnormally. They will produce abnormal group of cells which have no function of themselves, these cells utilize all the nutrient coming to bone marrow and supress the growth of normal blood cells. There will be decreased amount of normal blood cells-

Red blood cells- They contain hemoglobin which is required for carrying oxygen to other tissues, when they decrease in number there will be low tissues oxygenation and patient will feel weakness, fatigue
White blood cells- their main function is to fight against infection, so there will be high incidence of various infections

The most common cause of death in AML is Systemic infection which is due to decreased normal White blood cells.

Platelets - their function is in blood coagulation, decrease in their number suggests bleeding disorders

Hemorrhage due to decreased platelets is 2nd most common cause of death in AML

why AML is so BAD?

Most of the people die within 5 weeks without the treatment.
The five year survival is only 27% in average.
No specific cure is available

The American Cancer Society’s estimates for leukemia in the United States for 2017 are:
About 62,130 new cases of leukemia (all kinds) and 24,500 deaths from leukemia (all kinds)
About 21,380 new cases of acute myeloid leukemia (AML). Most will be in adults.
About 10,590 deaths from AML. Almost all will be in adults.[1]

What causes AML and what are the types?

The main pathology is the mutation of the normal stem cells into the abnormal ones

AML with genetic abnormality- Due to genetic predisposition
AML with myelodysplasia
Therapy related - Chloramphenicol, anticancer drugs like topoisonerase inhibitors
Radiation induced
Downs syndrome

How is the drug liposomal daunorubicin-cytarabine ?

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Normal daunorubicin-cytarabine combination has been used for many years for different types of AML, the new liposomal drug is coated with liposomal membrane capsule which helps in higher efficacy of drug.The liposomal membrane encapsulated drug is selectively uptaken by cancer cells which increases the efficacy.

The side effects

these are common side effect of this drug combination which is seen in more than 25% of the patients taking it.

Hemorrhages- bleeding gums, gastrointestinal bleeding,
fever, rashes
Nausea and vomiting, diarrhea/constipation,
Muscle and bone pain, weakness, fatigue
Respiratory difficulty, cough,pneumonia
Disturbances in sleep

My Comment on Approval of this drug

It is the first drug specifically approved for therapy related and myelodysplastic AML. The trial showed much significant improvement with new liposomal combination than the previous normal combination(10 month mean survival with liposomal vs 6 month survival with previous regimen in patient with therapy related and myelodysplastic AML) which is a good sign. Side effects are same with this and the previous combination.It was seen only in the trials, lets see how it goes in the overall patients of therapy related and myelodysplastic AML.

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