Recruitment of infants with sickle cell anemia to a Phase III trial: data from the BABY HUG study.
BACKGROUND
Protocol-eligible subjects may not be candidates for research participation or may decline. To determine factors that affected accrual, we evaluated enrollment in BABY HUG, a multi-center, randomized, placebo-controlled Phase III trial of hydroxyurea (HU) in infants with sickle cell anemia.


METHODS
An anonymized registry of potential subjects served as the primary source of data. Study coordinators considered all infants less than age 18 months with a hemoglobin FS diagnosis on newborn screening. Data included the number of potentially eligible subjects, whether parents were approached, and reasons for participating or declining.


RESULTS
Of 1106 potential participants, 28% were not approached for reasons such as prior poor adherence to clinical care. Interested families expressed willingness to contribute to medical knowledge (51%), hope of being randomized to receive hydroxyurea (51%), and desire for closer clinical care (51%) as reasons for participating. Disease severity or the perception that their child was ill had less impact on willingness to participate (16%). Parents who declined cited fear of research (19%), transportation problems (14%), and the demanding nature of the study (25%). Ultimately, 234 (21%) gave informed consent, with little variability of acceptance rates among sites. Importantly, the number of subjects enrolled correlated with the number of families that were approached. Sites that excluded patients based on clinical/psychosocial biases were not more successful in recruiting than those who approached all eligible subjects.


CONCLUSIONS
Large, demanding clinical trials require an adequate pool of potential participants. Approaching all potentially eligible patients without predetermined biases enhances success in recruitment.
Wynn
Lynn
L
St Jude Children's Research Hospital, Memphis, TN, United States. lynn.wynn@stjude.org
Miller
Scott
S
Faughnan
Lane
L
Luo
Zhaoyu
Z
Debenham
Ellen
E
Adix
Lea
L
Fish
Billie
B
Hustace
Tally
T
Kelly
Tracy
T
Macdermott
Marylou
M
Marasciulo
Joan
J
Martin
Brenda
B
McDuffie
Jennifer
J
Murphy
Mary
M
Rackoff
Betsy
B
Reed
Caroline
C
Seaman
Phillip
P
Thomas
Glenda
G
Wang
Winfred
W
D000755:Anemia, Sickle Cell / Q000175:diagnosis / Q000188:drug therapy; D000986:Antisickling Agents / Q000627:therapeutic use; D001294:Attitude to Health; D006801:Humans; D006918:Hydroxyurea / Q000627:therapeutic use; D007223:Infant; D007231:Infant, Newborn; D009042:Motivation; D015997:Neonatal Screening; D010290:Parents; D018579:Patient Selection
D000986:Antisickling Agents; D006918:Hydroxyurea
BMC Pediatr. 2007 Mar 09;7:12
17349034
Pediatrics. 2003 May;111(5 Pt 1):1037-41
12728085
J Pediatr Hematol Oncol. 2008 Nov;30(11):823-8
18989159
Cancer Nurs. 2000 Dec;23(6):444-51; quiz 451-2
11128123
J Pediatr. 2003 May;142(5):554-9
12756389
Anesthesiology. 2003 Oct;99(4):819-25
14508312
Lancet Oncol. 2006 Feb;7(2):141-8
16455478
Health Technol Assess. 2007 Nov;11(48):iii, ix-105
17999843
Pediatr Blood Cancer. 2010 Jul 15;55(1):129-33
20213846
Pediatr Blood Cancer. 2010 Feb;54(2):250-5
19731330
Am J Surg. 2007 Jan;193(1):32-9; discussion 40
17188084
J Clin Oncol. 2001 Mar 15;19(6):1728-33
11251003
Cancer. 2008 Jan 15;112(2):228-42
18008363
Res Nurs Health. 2006 Jun;29(3):172-5
16676344
Nurs Outlook. 2000 Sep-Oct;48(5):230-7
11044298
Pediatr Blood Cancer. 2010 Feb;54(2):260-4
19856395