Table 1

HINE-2 evolution of the patients according to SMN2 copy number and age of nusinersen initiation

One copy of SMN2
Two copies of SMN2
Three copies of SMN2
Sex, M:F, n18:221:010:157:7
Age at symptom onset3.0 (0.0–6.0)0.02.0 (0.0–6.0)4.5 (1.0–6.0)
Age at nusinersen initiation20.0 (0.35–294.0)2.019.0 (0.35–140.0)25.3 (4.0–294.0)
Disease duration before nusinersen17.5 (0.0–291.5)2.016.0 (0.35–140.0)17.5 (0.0–291.5)
HINE-2 score
M00.0 (0.0–4.0), n=370.0, n=10.0 (0.0–2.0), n=242.0 (0.0–4.0), n=12
M62.5 (0.0–17.0), n=344.0, n=11.0 (0.0–11.0), n=224.0 (1.0–17.0), n=11
M104.0 (0.0–24.0), n=325.0, n=12.5 (0.0–20.0), n=204.0 (1.0–24.0), n=11
Time effect p value<0.001/<0.0010.002
Delta M6M02.0 (0.0–16.0), p<0.0014.01.0 (0.0–11.0), p=0.0022.0 (1.0–16.0), p=0.019
Delta M10M03.0 (0.0–20.0), p<0.0015.02.5 (0.0–20.0), p=0.0033.0 (1.0–20.0), p=0.010
HINE-2 score (age ≤7 months old at onset of treatment)
M00.0 (0.0–4.0), n=13
M66.0 (2.0–16.0), n=11
M1011.0 (3.0–24.0), n=11
Time effect p value<0.001
Delta M6M06.0 (2.0–12.0), p<0.001
Delta M10M09.0 (3.0–20.0), p<0.001
HINE-2 score (age ≤2 years old at onset of treatment)
M00.0 (0.0–4.0), n=22
M65.0 (0.0–17.0), n=19
M109.0 (0.0–24.0), n=18
Time effect p value<0.001
Delta M6M04.0 (0.0–16.0), p<0.001
Delta M10M07.5 (0.0–20.0), p<0.001
HINE-2 score (age >2 years old at onset of treatment)
M00.0 (0.0–2.0), n=15
M61.0 (0.0–4.0), n=15
M100.5 (0.0–7.0), n=14
Time effect p value0.002
Delta M6M01.0 (0.0–2.0), p=0.003
Delta M10M00.5 (0.0–5.0), p=0.013
  • * Data are listed as median (range) in months.

  • ‡ One-way repeated measures ANOVA.

  • § Paired t-test.

  • ¶ Multiple regression demonstrated that there was significant difference in the changes of the HINE-2 scores, delta M6M0 and delta M10M0, between the two groups with two and three SMN2 copies with the standard coefficients β of 0.263 (p=0.003).

  • † Three patients were not included in the HINE-2 M0 analysis, due to the following reasons: one patient did not have baseline HINE-2 performed; two patients did not have HINE-2 performed.

  • HINE-2, Hammersmith Infant Neurologic Examination Part 2 motor milestones score; M0, before treatment; M6, 6 months of treatment; M10, 10 months of treatment; SMN, survival motor neuron.