New Guidelines Issued For Non-Transfusion-Dependent Thalassaemia

Date: Jul-16-2013STOCKHOLM - The Thalassaemia International Federation (TIF) has issued guidelines that aim to provide physicians with simple protocols intended to improve the detection and management of non-transfusion- dependent Thalassaemia (NTDT).

The guidelines are "the first of a kind," as earlier published recommendations have addressed the management of Thalassaemia as one clinical entity even though it has long been known that Thalassaemia has a wide spectrum of severity.

The new recommendations advocate the initiation of iron chelation therapy in NTDT patients with a liver iron concentration (LIC) ≥ 5 mg Fe/g dry weight, with therapy continued until patients reach levels

Patients who achieve this target should discontinue iron chelation therapy with an option for a "drug vacation."

Dr. Androulla Eleftheriou, who is executive director of the TIF, said that the guidelines were formulated by organization members from 112 national Thalassaemia patient associations in 62 countries worldwide and thus
represent a consensus of international expert option.

Because of migration and global population movements, NTDT exists in almost every region of the world, she added.

The new recommendations call for conscientious patient monitoring with LIC assessment every one to two years in order to identify individuals who are approaching values ≥ 5 mg Fe/g dry weight and thus need to start iron chelation therapy.

Concomitant serial measurement of serum ferritin levels every three months is recommended. In fact, serum ferritin levels should be considered the primary index of iron overload status when LIC measurement is not available.

The guidelines also state that the use of other conventional or experimental iron studies as well as clinical indicators of the severity of ineffective erythropoiesis may be used to support interpretation of serum ferritin level when it is the sole index of iron overload status.

Oral formulations of iron chelation therapy have been shown to improve quality of life and should be considered first for NTDT patients who require iron chelation therapy. A course of prolonged infusions with desferrioxamine can negatively impact quality of life and compliance, the latter of which has been shown to correlate negatively with survival.

Patients with NTDT do not require lifelong regular transfusions for survival although they may need occasional or even frequent transfusions in certain clinical settings and for defined periods of time. There are three clinically distinct forms of NTDT: Thalassaemia intermedia, HbE/Thalassaemia, and α-Thalassaemia.

Written by Jill Stein

Jill Stein is a Paris-based freelance medical writer.

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