New therapies in XXXX .

@entity1 @entity8561 @entity417 @entity19 @entity3458 @entity842 @entity369
CONTEXT: New therapies have evolved from our improved understanding of the biology of @entity3458 ( @entity3458 ) and the availability of a useful transgenic animal model. Several therapeutic options are available that interrupt the sickling process at various key pathways. @entity842 (NO)is a critical factor in the pathophysiology of @entity3458 and is a promising antisickling agent with vasodilation properties. NO regulates blood vessel tone, endothelial adhesion, and the severity of ischaemia-reperfusion injury and @entity417 in @entity3458 . Although NO is difficult to administer, its precursor, @entity8561 , is an oral supplement. STARTING POINT: J R Romero and colleagues recently demonstrated in sickle @entity19 that oral @entity369 supplementation induced NO production and reduced red-cell density by inhibiting the Gardos channel, which modulates cell hydration and polymerisation of haemoglobin S (Blood 2002; 99:1103-08). Haemoglobinopathies can be cured by stem-cell transplantation. This therapy is now accepted treatment in symptomatic @entity1 . However, most @entity1 lack a genotypically identical family donor. G La Nasa and colleagues demonstrated unrelated-donor stem-cell transplantation may give similar results to related-donor stem-cell transplantation when extended phenotypic matching is used (Blood 2002; 99: 4350-56). This pilot study offers the possibility of cure to @entity1 without a family donor. WHERE NEXT: Although potential opportunities to prevent morbidity in @entity3458 through new therapies are exciting, most @entity1 do not have access to standard multidisciplinary specialty care. @entity1 require both.
