Clinical Trial: Evaluation of the Efficacy in Decreasing Iron Absorption in Patients With Congenital Dyserythropoietic Anemia Type I by Treatment With LOSEC

Study Status: Recruiting
Recruit Status: Unknown status
Study Type: Interventional



Official Title:

Brief Summary:

Congenital Dyserythropoietic Anemia Type I (CDAI) is a recessive autosomal disease caused by ineffective erythropoiesis that causes Anemia & accumulation of iron due to increased absorption of iron in the intestine.

The iron is being accumulated in the body & causes damage of the liver, heart & endocrine glands.

The standard follow up of iron levels is done by ferritin blood test & although the test is not accurate it is the most available.

Medical treatment to removal of iron excess from the body is given in ferritin levels of 500-1000.

3 drugs are approved in the market:

  • Deferoxamine, given subcutaneous during the night, 5-7 nights/week and therefore is less used today.
  • Deferiprone-given 3 times a day, is a weaker chelator, although it seems like it is good to give it in combination with one of the 2 other drugs because it removes the iron from the heart's cells better.

A rare but severe adverse effect is Agranulocytosis. This drug is usually not given in childbirths.

-The 3rd drug which is mostly in use today is Deferasirox, given once daily, but has also adverse effects, among them- damage of liver & kidney function, damage to the digestive system, hearing and seeing.

Iron is being absorbed in acidic area, & thus drugs which decrease the acidity like hydrogen pump's inhibitors, can inhibit the absorption of iron, and indeed there are reports that these drugs decrease the absorption of iron and were used as treatment to hemochrom