S. Charache, EXPERIMENTAL-THERAPY OF SICKLE-CELL DISEASE - USE OF HYDROXYUREA, The American journal of pediatric hematology/oncology, 16(1), 1994, pp. 62-66
Purpose: Therapy of sickle cell disease with hydroxyurea is experiment
al. Patients and Methods: We have begun a randomized blinded clinical
trial to determine its clinical utility. The efficacy of this drug is
unproved and its risks, which include mutagenesis, teratogenesis and c
arcinogenesis, are poorly understood. These risks are explicitly state
d in our consent forms. A significant number of patients who are asked
to enroll refuse to enter the study. This refusal is probably because
of individual variations in perception of risk and personal inconveni
ence, as well as differences in perception of personal benefit. We hav
e a few hints as to which patients are more likely to produce increase
d amounts of fetal hemoglobin, but our findings do not indicate which
patients are most likely to show a good clinical-response. Results: Ou
r study group decided not to treat patients under 18 years of age with
hydroxyuria until clinical efficacy of the drug is proved in adults.
We have criteria for selecting patients for entry into our ongoing stu
dy, but the criteria are based more on study design than on an estimat
e of present or future severity of the manifestations of sickle cell d
isease. Conclusions: Features of our previous study and results of the
present trial may be helpful in defining indications for bone marrow
transplantation in children with sickle cell disease.