Table 2.

Change in TRV and laboratory and clinical markers over the 2-y study period

Group 1Group 2Group 3Group 4All
Mean changeP*Mean changeP*Mean changeP*Mean changeP*Mean changeP*
TRV, m/s 0.02 (0.40) .96 −0.14 (0.33) .02* −0.11 (0.35) <.01† −0.01 (0.30) .82 −0.06 (0.36) <.01* 
TRV ≥2.5 m/s, n (%) 0 (0.00) >.99 5 (14.71) .17 8 (11.11) .10 0 (0.00) >.99 13 (6.37) .12 
MCV, fL −1.30 (3.72) <.01† 14.26 (9.09) <.0001† −1.30 (9.3) .64 −0.42 (3.10) .41 1.51 (9.13) .13 
HbF, % −2.37 (3.54) <.0001† 10.11 (6.31) <.0001† −1.71 (5.42) .01† −0.28 (2.10) .68 0.48 (6.45) .77 
Hb, g/dL 0.19 (0.68) .08 1.02 (1.04) <.0001† 0.10 (0.88) .36 0.06 (0.83) .65 0.28 (0.91) <.001† 
ARC, ×109/L 0.00 (0.07) .66 −0.13 (0.11) <.0001† 0.01 (0.08) .37 0.02 (0.10) .33 −0.02 (0.10) .03 
LDH, U/L −12.78(91.09) .29 −182.75 (150.92) <.0001† −50.19 (196.5) .02 17.93 (169.06) .52 −49.27 (170.68) <.0001† 
Bilirubin, mg/dL 0.23 (0.88) .01 −1.09 (1.21) <.0001† 0.01 (1.16) .69 0.77 (1.40) <.01 0.03 (1.28) .79 
WBC, ×109/L −0.14 (3.42) .97 −3.95 (4.98) <.0001† 0.30 (3.03) .40 1.09 (4.26) .13 −0.41 (4.10) .38 
Platelets, ×109/L 3.22 (77.06) .55 −29.29 (146.16) .06 46.61 (170.54) <.01† −22.28 (102.58) .01 8.19 (135.14) .81 
VWF, % 7.52 (25.4) <.01 −23.30 (32.21) <.001† 2.90 (35.77) .55 14.73 (41.20) .11 0.72 (34.87) .83 
FVIII, % 15.08 (46.23) .02 −8.86 (42.44) .24 10.77 (50.19) .12 5.41 (57.58) .66 7.48 (48.84) .05 
CRP, mg/dL −0.01 (0.65) .37 −0.38 (1.36) .24 0.08 (0.73) .99 0.04 (0.23) .44 −0.05 (0.84) .76 
Ferritin, ng/mL −4.75 (232.29) .06 −10.40 (121.02) .62 16.50 (380.26) .17 −18.72 (1997.42) .59 −1.01 (894.75) .75 
E-selectin 2.20 (41.35) .75 −69.20 (63.22) <.001† −4.82 (34.4) .39 −6.15 (26.65) .37 −13.92 (48.85) <.01† 
sVCAM −69.33 (271.66) .18 −312.03 (409.08) <.01† −188.43 (443.89) <.001† 61.26 (335.69) .87 −133.99 (385.52) <.0001† 
EGF 27.07 (186.32) .39 −47.73 (114.59) .09 −3.91 (164.54) .74 68.08 (302.16) .56 9.25 (191.43) .83 
IL-7 −0.44 (12.01) .67 −2.85 (9.88) .31 −8.27 (26.35) <.01† −1.30 (8.93) .21 −3.72 (18.00) <.01† 
MIP-1α −12.01 (107.38) .45 60.58 (1114.29) .18 −139.31 (649.01) .10 −68.58 (259.56) .21 −52.04 (608.13) <.01† 
MIP-1β 8.75 (63.74) .58 −17.90 (44.03) .10 −15.87 (51.44) .09 −4.92 (34.48) .94 −6.48 (53.32) .10 
sCD40L −1189.1 (7659.9) .38 −8280.4 (9301.3) <.01† −494.6 (6620.05) .63 −1606.8 (8174.92) .75 −2244.4 (8102.34) <.01† 
IFN-α2 19.65 (78.35) .30 −6.82 (34.59) .13 −35.06 (164.91) <.01† −2.25 (28.81) .76 −7.25 (110.17) .17 
IL-4 3.58 (19.39) .49 0.63 (9.26) .94 −6.16 (19.31) .03 −0.54 (9.49) .82 −0.94 (17.13) .27 
NT-proBNP, pg/mL −33.60 (100.41) .03 −13.93 (159.38) .04 −28.19 (86.48) .03 −42.45 (95.72) <.05 −29.34 (108.58) <.0001† 
UPCR −17.04 (106.19); P = .16 −36.15 (80.61); <.01† −17.40 (184.03); .01† −21.86 (155.29); P = .49 −21.36 (143.74); <.001† 
ACS‡, event counts 0.07 (0.45); P = .39 −0.29 (0.52); <.01† −0.11 (0.83); P = .17 −0.05 (0.39); P = .69 −0.08 (0.62); P = .03 
VOC‡, event counts 0.12 (1.11); P = .53 −0.06 (2.01); P = .79 0.04 (1.76); P = .55 −0.28 (0.75); P = .03 −0.01 (1.48); P = .74 
ACS + VOC‡, event counts 0.19 (1.15); P = .32 −0.35 (2.01); P = .31 −0.07 (2.27); P = .99 −0.33 (0.86); P = .03 −0.09 (1.73); P = .53 
Group 1Group 2Group 3Group 4All
Mean changeP*Mean changeP*Mean changeP*Mean changeP*Mean changeP*
TRV, m/s 0.02 (0.40) .96 −0.14 (0.33) .02* −0.11 (0.35) <.01† −0.01 (0.30) .82 −0.06 (0.36) <.01* 
TRV ≥2.5 m/s, n (%) 0 (0.00) >.99 5 (14.71) .17 8 (11.11) .10 0 (0.00) >.99 13 (6.37) .12 
MCV, fL −1.30 (3.72) <.01† 14.26 (9.09) <.0001† −1.30 (9.3) .64 −0.42 (3.10) .41 1.51 (9.13) .13 
HbF, % −2.37 (3.54) <.0001† 10.11 (6.31) <.0001† −1.71 (5.42) .01† −0.28 (2.10) .68 0.48 (6.45) .77 
Hb, g/dL 0.19 (0.68) .08 1.02 (1.04) <.0001† 0.10 (0.88) .36 0.06 (0.83) .65 0.28 (0.91) <.001† 
ARC, ×109/L 0.00 (0.07) .66 −0.13 (0.11) <.0001† 0.01 (0.08) .37 0.02 (0.10) .33 −0.02 (0.10) .03 
LDH, U/L −12.78(91.09) .29 −182.75 (150.92) <.0001† −50.19 (196.5) .02 17.93 (169.06) .52 −49.27 (170.68) <.0001† 
Bilirubin, mg/dL 0.23 (0.88) .01 −1.09 (1.21) <.0001† 0.01 (1.16) .69 0.77 (1.40) <.01 0.03 (1.28) .79 
WBC, ×109/L −0.14 (3.42) .97 −3.95 (4.98) <.0001† 0.30 (3.03) .40 1.09 (4.26) .13 −0.41 (4.10) .38 
Platelets, ×109/L 3.22 (77.06) .55 −29.29 (146.16) .06 46.61 (170.54) <.01† −22.28 (102.58) .01 8.19 (135.14) .81 
VWF, % 7.52 (25.4) <.01 −23.30 (32.21) <.001† 2.90 (35.77) .55 14.73 (41.20) .11 0.72 (34.87) .83 
FVIII, % 15.08 (46.23) .02 −8.86 (42.44) .24 10.77 (50.19) .12 5.41 (57.58) .66 7.48 (48.84) .05 
CRP, mg/dL −0.01 (0.65) .37 −0.38 (1.36) .24 0.08 (0.73) .99 0.04 (0.23) .44 −0.05 (0.84) .76 
Ferritin, ng/mL −4.75 (232.29) .06 −10.40 (121.02) .62 16.50 (380.26) .17 −18.72 (1997.42) .59 −1.01 (894.75) .75 
E-selectin 2.20 (41.35) .75 −69.20 (63.22) <.001† −4.82 (34.4) .39 −6.15 (26.65) .37 −13.92 (48.85) <.01† 
sVCAM −69.33 (271.66) .18 −312.03 (409.08) <.01† −188.43 (443.89) <.001† 61.26 (335.69) .87 −133.99 (385.52) <.0001† 
EGF 27.07 (186.32) .39 −47.73 (114.59) .09 −3.91 (164.54) .74 68.08 (302.16) .56 9.25 (191.43) .83 
IL-7 −0.44 (12.01) .67 −2.85 (9.88) .31 −8.27 (26.35) <.01† −1.30 (8.93) .21 −3.72 (18.00) <.01† 
MIP-1α −12.01 (107.38) .45 60.58 (1114.29) .18 −139.31 (649.01) .10 −68.58 (259.56) .21 −52.04 (608.13) <.01† 
MIP-1β 8.75 (63.74) .58 −17.90 (44.03) .10 −15.87 (51.44) .09 −4.92 (34.48) .94 −6.48 (53.32) .10 
sCD40L −1189.1 (7659.9) .38 −8280.4 (9301.3) <.01† −494.6 (6620.05) .63 −1606.8 (8174.92) .75 −2244.4 (8102.34) <.01† 
IFN-α2 19.65 (78.35) .30 −6.82 (34.59) .13 −35.06 (164.91) <.01† −2.25 (28.81) .76 −7.25 (110.17) .17 
IL-4 3.58 (19.39) .49 0.63 (9.26) .94 −6.16 (19.31) .03 −0.54 (9.49) .82 −0.94 (17.13) .27 
NT-proBNP, pg/mL −33.60 (100.41) .03 −13.93 (159.38) .04 −28.19 (86.48) .03 −42.45 (95.72) <.05 −29.34 (108.58) <.0001† 
UPCR −17.04 (106.19); P = .16 −36.15 (80.61); <.01† −17.40 (184.03); .01† −21.86 (155.29); P = .49 −21.36 (143.74); <.001† 
ACS‡, event counts 0.07 (0.45); P = .39 −0.29 (0.52); <.01† −0.11 (0.83); P = .17 −0.05 (0.39); P = .69 −0.08 (0.62); P = .03 
VOC‡, event counts 0.12 (1.11); P = .53 −0.06 (2.01); P = .79 0.04 (1.76); P = .55 −0.28 (0.75); P = .03 −0.01 (1.48); P = .74 
ACS + VOC‡, event counts 0.19 (1.15); P = .32 −0.35 (2.01); P = .31 −0.07 (2.27); P = .99 −0.33 (0.86); P = .03 −0.09 (1.73); P = .53 

Data are mean (standard deviation) or n (%), except TRV ≥2.5 m/s is presented as n (%). Change between baseline and year 2 follow-up was calculated and tested from a difference of 0 using the 1-sample Student t test or the Wilcoxon signed-rank test for continuous variables and McNemar’s test for categorical variable.

Group 1: not on disease modifying therapy at baseline and 2-y evaluation; group 2: not on disease modifying therapy at baseline evaluation, but initiated  hydroxyurea prior to the 2-y evaluation; group 3: on hydroxyurea at both baseline and 2-y evaluation; group 4: on monthly blood transfusions at both baseline and 2-y evaluation. FVIII, factor VIII; VOC, vaso-occlusive (acute pain) crisis; VWF, von Willebrand factor.

*

Raw P value.

†

PFDR < .05.

‡

Mean change in event counts between the 2-y interval prior to baseline and the 2-y study period.

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