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Foreword – European Haematology, 2008;2(1):6

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Published Online: Aug 22nd 2011 European Haematology, 2008;2(1):6
Authors: Luigi D Notarangelo
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This issue of European Haematology covers several topics that are of great interest to haematologists because they highlight recent advances in the field or discuss current controversies in the managament of patients.

This issue of European Haematology covers several topics that are of great interest to haematologists because they highlight recent advances in the field or discuss current controversies in the managament of patients.
While inherited bleeding disorders are generally rare, some (such as haemophilia and von Willebrand disease [VWD]) occur more frequently. Most importantly, there is a need to streamline strategies that may help identify patients who are at significant risk of bleeding. The recent development of a bleeding severity score (BSS) may be helpful, as indicated by preliminary experiences. It will be important to reproduce these findings in heterogeneous populations, and to investigate the potential benefit of this approach in children as well as in adults.
Following encouraging results obtained in haemophilia, prophylaxis has recently also been attempted in VWD. While such an approach should be used only in patients at high risk of bleeding, its cost-effectiveness has yet to be firmly established, and additional studies are therefore needed. There have also been significant advances in the characterisation of the molecular and cellular bases of congenital disorders of platelet function, and these findings are illustrated in this issue.
Acquired platelet disorders, in particular chronic idiopathic thrombocytopenic purpura (cITP), remain a matter of concern for many patients worldwide. In the past, patients with severely symptomatic cITP who failed treatment with corticosteroids were eventually treated using splenectomy. Medical management of cITP has gained in importance in recent years. One such new drug, romiplostim, has an overall response rate as high as 80%. Long-term evaluation of its efficacy and surveillance studies are of paramount importance in order to better define its role in the treatment of cITP.
Refractory ITP of childhood poses significant problems both in the diagnostic approach and in the identification of optimal methods of treatment. Importantly, guidelines and recommendations are not always followed in clinical practice. It can be expected that the Paediatric and Adult Registry of Chronic ITP will help to divide this heterogeneous cohort of patients into more homogeneous groups so that robust prospective clinical studies can be performed in this area.
In several countries, newborn screening programmes (NBS) are expanding to include a larger number of disorders. Implementation of a sickle-cell disease (SCD) NBS was recommended in the US as early as 20 years ago. A targeted screening approach was followed by unviersal screening. Other haemoglobinopathies could also be considered, although in these cases early identification of the disease status would provide little advantage to the affected child. In general, it is important that any NBS be associated with a solid plan that includes confirmatory testing and clear indications for and easy access to treatment.
These are just some of many interesting topics that are presented in this issue of European Haemaology. As always, discussing controversial topics is the best way to achieve consensus and reach the next level in the diagnosis and management of human diseases. ■

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