Primary stroke prevention in Nigerian children with sickle cell disease (SPIN): challenges of conducting a feasibility trial.
BACKGROUND
The majority of children with sickle cell disease (SCD), approximately 75%, are born in sub-Saharan Africa. For children with elevated transcranial Doppler (TCD) velocity, regular blood transfusion therapy for primary stroke prevention is standard care in high income countries, but is not feasible in sub-Saharan Africa.


METHODS
In the first U.S. National Institute of Health (NIH) sponsored SCD clinical trial in sub-Saharan Africa, we describe the protocol and challenges unique to starting a clinical trial in this region. We are conducting a single arm pilot trial of hydroxyurea therapy in children with TCD velocity ≥200 cm/sec in the middle cerebral arteries. Eligible children will be placed on hydroxyurea (n = 40) and followed for 3 years at Aminu Kano Teaching Hospital, Nigeria. Adherence will be measured via the Morisky Scale and adverse events will be determined based on hospitalization.


RESULTS
Originally, a randomized placebo trial was planned; however, placebo was not approved by the local Ethics Committee. Hence a single arm trial of hydroxyurea will be conducted and five controls per patient with normal TCD measurements will be followed to compare the rate of adverse events to those with abnormal TCD measurements taking hydroxyurea. Using non-NIH funding, over 9 months, multiple face-to-face investigator meetings were conducted to facilitate training.


CONCLUSIONS
A hydroxyurea trial (NCT01801423) for children with SCD is feasible in sub-Saharan Africa; however, extensive training and resources are needed to build a global patient oriented multi-disciplinary research team with a common purpose.
Department of Haematology and Blood Transfusion, Bayero University/Aminu Kano Teaching Hospital, Kano, Nigeria.
Najibah A
NA
Galadanci
Shehu U
SU
Abdullahi
Musa A
MA
Tabari
Shehi
S
Abubakar
Raymond
R
Belonwu
Auwal
A
Salihu
Kathleen
K
Neville
Fenella
F
Kirkham
Baba
B
Inusa
Yu
Y
Shyr
Sharon
S
Phillips
Adetola A
AA
Kassim
Lori C
LC
Jordan
Muktar H
MH
Aliyu
Brittany V
BV
Covert
Michael R
MR
DeBaun
D000755:Anemia, Sickle Cell / Q000150:complications / Q000000981:diagnostic imaging / Q000188:drug therapy; D000986:Antisickling Agents / Q000008:administration & dosage*; D002648:Child; D002675:Child, Preschool; D005240:Feasibility Studies; D005260:Female; D005500:Follow-Up Studies; D019983:Guideline Adherence; D006801:Humans; D006918:Hydroxyurea / Q000008:administration & dosage*; D008297:Male; D020768:Middle Cerebral Artery / Q000000981:diagnostic imaging; D009549:Nigeria; D010865:Pilot Projects; D020521:Stroke / Q000000981:diagnostic imaging / Q000517:prevention & control; D017585:Ultrasonography, Doppler, Transcranial / Q000379:methods
D000986:Antisickling Agents; D006918:Hydroxyurea
Nigeria; hydroxyurea; primary stroke prevention; sickle cell disease