Iron-chelating therapy and the treatment of thalassemia

Aims

To assess the natural history, risk factors for death and deterioration of patients with Eisenmenger Syndrome.

Methods

The clinical course of 188 patients from three different cardiac centres specialized in adult congenital heart defects, followed for a median period of 31 years, was retrospectively analysed. According to the diagnosis, 64 males and 124 females, mean age at last follow-up 33·02±12·8 years, were divided into two groups: simple (128) and complex (60) congenital heart disease. Events analysed were: time variation of Ability Index, pulmonary and cerebral complications, non-cardiac surgery, urate metabolism and renal function, arrhythmias, pregnancy and contraception, medical therapy and transplantation, death from all causes and sudden death.

Results

Eighty-four percent of the overall population showed a satisfactory Ability Index (1 and 2) at the first attendance (median age 25 years). Patients with complex heart disease and Eisenmenger reaction had earlier clinical deterioration (18·6±11·3 vs 26·7±12·2) and shorter survival (25·8±7·9 vs 32·5±14·6 years). Thirty-eight (20·2%) patients had at least one episode of haemoptysis between 20 and 40 years of age but it did not modify overall survival; 25 (13·2%) had pulmonary thromboembolism at a mean age of 35·2±13·4 years. Fifteen (7·9%) had a stroke and 7 (3·7%) a cerebral abscess at a mean age of 31·4±15·7 and 24·1±4·9 years, respectively. Cerebral complications influenced the quality of life but did not modify survival. Patients who had venesection showed a 2·04 times greater hazard ratio for haemoptysis. Venesection did not reduce cerebral complications and in 20% caused anaemia and iron deficiency. Other non-cardiac surgery with general anaesthesia carried risks (23·5% of deaths). Significant maternal mortality (27%) in relation to pregnancy occurred with constant deterioration in physical status, high incidence of spontaneous abortions (35·8%) and cardiac abnormalities in offspring (20%). Sixty-one patients died during follow-up, mainly by sudden death (29·5%), heart failure (22·9%) and from haemoptysis (11·4%). Eight patients had heart and lung transplantation and five died 1 week to 4 years after transplant. Deterioration in Ability Index (worsening symptoms), age, complex defects, blood creatinine level, right ventricular dysfunction and non-cardiac surgery were variables which affected the prognosis adversely with uni- and multivariate analysis.

Conclusions

patients with Eisenmenger syndrome can survive to the seventh decade with informed medical care and protection from special risks

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Eisenmenger syndrome. Factors relating to deterioration and death

Polycythemia vera: myths, mechanisms, and management.

Cystic fibrosis in adults: 75 cases and a review of 232 cases in the literature

Traditionally, polycythaemia has been used to identify a group of varied disorders with an increase in circulating red cells that are typified by a persistently raised haematocrit (Hct). Since only the red cell lineage is involved, the term erythrocytosis has more validity and will be used throughout this article. Polycythaemia will be retained in relation to the clonal disorder, polycythaemia vera (PV), in which three cell lineages are involved.

https://onlinelibrary.wiley.com/doi/pdf/10.1111/j.1365-2141.2005.05535.x

Guidelines for the diagnosis, investigation and management of polycythaemia/erythrocytosis

New approaches to the transfusion management of thalassemia

Acute reduction in red cell volume (RCV) without significant alterations of blood volume in 22 patients with severe polycythemia secondary to cyanotic congenital heart disease resulted in a decrease in peripheral vascular resistance and an increase in stroke volume, systemic blood flow (SBF), and systemic oxygen transport. These changes are probably related to the decreased blood viscosity and yield shear stress associated with lower red cell concentrations. Hypervolemia in hypoxic polycythemia should be maintained in order to sustain an adequate SBF. In contrast to acute phlebotomy which may be expected to decrease blood oxygen content and SBF, the replacement of whole blood with plasma or 5% albumin is shown to result in an increased systemic blood flow and oxygen delivery.

Acute Hemodynamic Effects of Red Cell Volume Reduction in Polycythemia of Cyanotic Congenital Heart Disease

Simultaneous red cell volume and plasma volume determinations were performed in 20 patients with cyanotic congenital heart disease. Significant hypervolemia was shown to result from an increased red cell volume. Plasma volume may be reduced but usually remains within the normal range. The good correlation between the venous hematocrit and red cell volume per kilogram provides the basis for the estimation of the red cell volume from the venous hematocrit. The common clinical practice of estimating the degree of hypoxemia from the red cell volume (or hematocrit) is at best very gross due to the large variability of the red cell volume at any given level of arterial oxygen saturation in patients with cyanotic heart disease. Red cell volume and plasma volume should be independently and simultaneously measured if accurate estimates of total blood volume changes are desired. Blood volume measurements serve as an index and guide in the medical and surgical treatment of polycythemic cyanotic patients.

Lawrence N. Button

Papers

New approaches to the transfusion management of thalassemia

Acute Hemodynamic Effects of Red Cell Volume Reduction in Polycythemia of Cyanotic Congenital Heart Disease

Blood volume changes in cyanotic congenital heart disease

Citrate-phosphate-dextrose solution for preservation of human blood: a further report.

Simultaneous determination of the volume of red cells and plasma for survival studies of stored blood.