Pablo Bartolucci1, Frédéric Galactéros. 1. Unité des Maladies Génétiques du Globule Rouge, Hôpital Henri-Mondor AP-HP, Université Paris Est, Créteil, Paris, France. pablo.bartolucci@hmn.aphp.fr
Abstract
PURPOSE OF REVIEW: This review provides an overview of the clinical management of sickle-cell disease (SCD) with recently published findings. RECENT FINDINGS: Unfortunately, negative observations did not confirm the hope that therapies acting on nitric oxide-cyclic GMP signaling, NSAIDs or Gardos channel inhibitor would control SCD vaso-occlusive crises. The safety of hydroxycarbamide was further supported by two observational studies covering 20 years and over 2 years in young children. Concerning the management of chronic visceral complications of SCD, the STOP II trial showed the risk of discontinuing blood exchange transfusion for children with transcranial Doppler-assessed accelerated blood-flow velocities. The French multicenter Etendard study found that only 25% of SCD patients with tricuspid regurgitation velocity (TRV) 2.5 m/s or more on echocardiograms had catheterization-confirmed pulmonary hypertension. However, elevated TRV, regardless of its cause, was associated with higher mortality. Finally, recent results identified new therapeutic strategies for the treatment and prevention of renal dysfunction, priapism and skin ulcers, but prospective studies are needed to confirm those findings. SUMMARY: SCD treatment relies on concomitant preventive and curative measures to control its acute and chronic manifestations. Pathophysiologic advances have enabled better management, with new therapeutics highly likely in the near future.
PURPOSE OF REVIEW: This review provides an overview of the clinical management of sickle-cell disease (SCD) with recently published findings. RECENT FINDINGS: Unfortunately, negative observations did not confirm the hope that therapies acting on nitric oxide-cyclic GMP signaling, NSAIDs or Gardos channel inhibitor would control SCD vaso-occlusive crises. The safety of hydroxycarbamide was further supported by two observational studies covering 20 years and over 2 years in young children. Concerning the management of chronic visceral complications of SCD, the STOP II trial showed the risk of discontinuing blood exchange transfusion for children with transcranial Doppler-assessed accelerated blood-flow velocities. The French multicenter Etendard study found that only 25% of SCDpatients with tricuspid regurgitation velocity (TRV) 2.5 m/s or more on echocardiograms had catheterization-confirmed pulmonary hypertension. However, elevated TRV, regardless of its cause, was associated with higher mortality. Finally, recent results identified new therapeutic strategies for the treatment and prevention of renal dysfunction, priapism and skin ulcers, but prospective studies are needed to confirm those findings. SUMMARY:SCD treatment relies on concomitant preventive and curative measures to control its acute and chronic manifestations. Pathophysiologic advances have enabled better management, with new therapeutics highly likely in the near future.
Authors: Amy Geard; Gift D Pule; Bernard Chetcha Chemegni; Valentina J Ngo Bitoungui; Andre P Kengne; Emile R Chimusa; Ambroise Wonkam Journal: Br J Haematol Date: 2017-05-03 Impact factor: 6.998
Authors: Geraldo B Silva Junior; Ana Patrícia F Vieira; Amanda X Couto Bem; Marília P Alves; Gdayllon C Meneses; Alice M C Martins; Sonia M H A Araújo; Alexandre V Libório; Elizabeth F Daher Journal: Int J Clin Pharm Date: 2014-06-17
Authors: Pablo Bartolucci; Anoosha Habibi; Thomas Stehlé; Gaetana Di Liberto; Marie Georgine Rakotoson; Justine Gellen-Dautremer; Sylvain Loric; Stéphane Moutereau; Dil Sahali; Orianne Wagner-Ballon; Philippe Remy; Philippe Lang; Philippe Grimbert; Etienne Audureau; Bertrand Godeau; Frédéric Galacteros; Vincent Audard Journal: J Am Soc Nephrol Date: 2015-11-19 Impact factor: 10.121