N-Acetylcysteine in Patients With Sickle Cell Disease (NAC).

The primary aim of this study is to evaluate the effect of the drug N-Acetylcysteine on the frequency of pain in daily life in patients with Sickle Cell Disease (SCD).



Pain is an invalidating hallmark of this disease and has a considerable impact on the Quality of Life of patients and the medical health care system. Oxidative stress is hypothesized to play a central role in its pathophysiology. In pilot studies the administration of N-Acetylcysteine (NAC) resulted in a reduction of oxidative stress. Moreover, administration of NAC seemed to decrease hospitalization for painful crises in a small pilot study in patients with SCD.



This study will be performed as a multicenter, randomized, controlled trial where patients will be treated with either NAC or placebo for a period of 6 months. We expect that NAC can reduce the frequency of pain in patients with SCD, thereby improving their quality of life and participation in society.

Our primary hypothesis is that the drug N-Acetylcysteine, a scavenger of free oxygen radicals and a precursor of glutathione, can reduce the frequency of daily life pain in patients with Sickle Cell Disease by reducing oxidative stress.

Evaluated over a 6 month intervention period with montly follow-up visits.

Experimental:

N-Acetylcysteine (N-Acetylcysteine 600mg 1 oral tablet twice daily during 6 months).



Placebo Comparator:

(Placebo 1 oral tablet twice daily during 6 months).