Role of Haematopoietic Stem Cell Transplantation in management of Severe Chronic Neutropenia.

Role of Haematopoietic Stem Cell Transplantation in management of Severe Chronic Neutropenia. Curr Drug Targets. 2018 Mar 08;: Authors: Fioredda F Abstract The inherited form of Severe Chronic Neutropenia called Severe Congenital Neutropenia (SCN) is an intrinsic defect of neutrophils maturation. SCN affected patients are at risk to develop lethal in-fections and to transform into myelodisplasia (MDS) and/or acute laeukemia(AL). Use of Granulocyte-Colony-Stimulating Factor (G-CSF) has significantly improved the prognosis of affected patients, but the only definitive cure for the disease is Haematopoietic Stem Cell Trans-plantation (HSCT). Overall Survival of SCN patients has been assessed at around 85% after 3 years from transplantation . The morbility and mortality associated to the procedure still represents a con-siderable portion, thus making the absolute indication to HSCT reserved for those SCN patients who have already transformed into MDS/AL or for those subjects who do not show a satisfying re-sponse to G-CSF. Indication to HSCT remains weak for those subjects who are easily managed with G-CSF at low/standard doses. Performing HSCT in patients who have a matched sibling donor or in those subjects harbouring "high risk mutations" is still a matter of discussion . Studies on SCN outcome with G-CSF management vs HSCT at the same disease's conditions will be necessary to tailor the most appropriate therapeutic choice for SCN aff...
Source: Current Drug Targets - Category: Drugs & Pharmacology Authors: Tags: Curr Drug Targets Source Type: research