Management of Cooley's anaemia

The current management of Cooley's anaemia has succeeded in prolonging the patient's life to at least the fourth decade, in allowing a number of the patients to procreate children, and in allowing most of them to lead a nearly unrestricted normal life. The price to be paid for this is the cumbersome and expensive adherence to a rigorous regimen of chronic transfusion and compliance with chelation therapy. Economical and psychological factors make it difficult for many patients to benefit from these advances, but those who can may be reasonably assured of a long and fruitful existence. It must be realized that the only defect in Cooley's anaemia is one of haemoglobin synthesis, and well-managed patients are in any other aspect capable of leading a normal life. In certain areas of the world, such as Cyprus (Loukopoulos et al, 1990) and Sardinia (Cao et al, 1990), extensive and intensive programmes of prenatal screening have reduced to nearly negligible levels the birth of children with Cooley's anaemia (see Chapter 9 this volume). Yet, in many countries a complete management of Cooley's anaemia is today not feasible for economic or cultural reasons, and this disease remains a serious public health problem. The future of the management of Cooley's anaemia appears at the moment full of hopes and frustrations (Gale, 1989; Bank, 1990). However, it is important to understand that if the current group of patients with Cooley's anaemia have to be the beneficiaries of future developments (be it genetic engineering and/or advances in histocompatibility matching), it is necessary that these patients be maintained in excellent clinical shape. It would be very sad when a cure for Cooley's anaemia became available, if most patients could not avail themselves of it because of pre-existing irreversible body damage.