Guidelines for the management of non-transfusion-dependent β-Thalassaemia
On the other hand, non-transfusion-dependent β-thalassaemia (NTDT) - the focus of these guidelines - is the term used for patients who do not require lifelong regular transfusions for survival, such as patients with β-thalassaemia intermedia or mild-moderate haemoglobin E/β-thalassaemia. It should b...
| Main Authors: | , , |
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| Corporate Author: | |
| Format: | eBook |
| Language: | English |
| Published: |
Nicosia, Cyprus
Thalassaemia International Federation
2023, 2023
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| Edition: | 3rd edition |
| Online Access: | |
| Collection: | National Center for Biotechnology Information - Collection details see MPG.ReNa |
| Summary: | On the other hand, non-transfusion-dependent β-thalassaemia (NTDT) - the focus of these guidelines - is the term used for patients who do not require lifelong regular transfusions for survival, such as patients with β-thalassaemia intermedia or mild-moderate haemoglobin E/β-thalassaemia. It should be stressed, however, that transfusion receipt is not always a measure of underlying disease severity but can also be attributed to access to blood products or reflect a physician or patient choice, with varying practices worldwide especially in patients with moderate phenotypes. In recent clinical trials, the use of the NTDT/TDT classification has commonly been associated with the patient's transfusion profile in the past six months (e.g., <6 red blood cell units denoting NTDT). This may be practical when taking immediate management decisions especially as relates to anaemia and iron overload. However, the natural course of a patient's disease course should always be taken into consideration since many patients with NTDT go on to become TDT following permanent morbidity development while also some patients with TDT can become NTDT following interventions that decrease transfusion requirement The thalassaemias are inherited haemoglobin disorders characterized by defective synthesis of the α-globin (α-thalassaemia) or β-globin (β-thalassaemia) chains of adult haemoglobin A. A variety of β-thalassaemia phenotypes can result from heterozygous, compound heterozygous, or homozygous inheritance of β-globin gene mutations, or their co-inheritance with structural haemoglobin variants such as haemoglobin E or other secondary genetic modifiers [1-5]. Transfusion-dependence has recently become an essential factor in classifying the various phenotypes of β-thalassaemia. Patients requiring lifelong regular transfusion therapy for survival are considered as having transfusion-dependent β-thalassaemia (TDT), such as patients with β-thalassaemia major or sever haemoglobin E/β-thalassaemia. |
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| Item Description: | Title from NCBI Bookshelf entry page |
| Physical Description: | 1 online resource illustrations |
| ISBN: | 9789963717255 |