N-Acetylcysteine in Patients With Sickle Cell Disease
N-Acetylcysteine in Patients With Sickle Cell Disease
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. The investigators expect
 that NAC can reduce the frequency of pain in patients with SCD, thereby improving their
 quality of life and participation in society.
Inclusion Criteria:

 -  Age 12 years or older

 -  Sickle cell disease, either homozygous sickle cell disease (HbSS), compound
 heterozygous sickle cell disease (HbSC), HbSβ0 or HbSβ+ thalassemia

 -  History of at least 1.0 painful crisis per year in the past 3 years (visit to medical
 facility is not required)

 Exclusion Criteria:

 -  Chronic blood transfusion or transfusion in the preceding 3 months

 -  Painful crisis in the last 4 weeks (with respect to the moment of inclusion)

 -  Pregnancy, breast feeding or the desire to get pregnant in the following 7 months

 -  Known active gastric/duodenal ulcers

 -  Hydroxycarbamide (HC) treatment with unstable dose in the last 3 months or started on
 HC shorter then 6 months prior to study

 -  Known poor compliance in earlier trials regarding the completion of pain diaries

 -  Insufficient compliance in run-in period

 -  Known hypersensitivity to acetylcysteine or one of the other components of the study
 medication

 -  Use of pain medication for sickle-cell related pains on more than 15 days per month in
 the past 6 months ('chronic pain').