Table 2 Side effects in medically treated patients with CHI: overview of side effects that occurred while on therapy with one or more of the analyzed medication. Side effects were linked to the corresponding dose that was applied. Note double entries of patients that experienced more than one side effect

mg/kg • d

DZX treated n = 644

hypertrichosis

n = 170 (52 %)

11.6 (±2.9), range 5–30 (n = 132)

side effects mentioned n = 325 (50 %)

fluid retention/electrolyte imbalances/edema

n = 99 (30 %)

12.3 (±2.8), range 8.5–17.5 (n = 48)

vomiting/poor appetite

n = 40 (12 %)

13.3 (±8.2), range 10–60 (n = 39)

bone marrow suppression

n = 11 (3 %)

10.4 (±0.46), range 10–10.8 (n = 4)

heart failure

n = 12 (3.7 %)

16 (±6.7), range 7.5–30 (n = 7)

anaphylactic reaction

n = 3 (0.9 %)

8.5 (n = 1)

renal failure

n = 2 (0.6 %)

60 (n = 1)

sinustachycardia

n = 1 (0.3 %)

up to 15 (n = 1)

no (major) side effects (specifically mentioned)

n = 39 (12 %)

13.2 (±2.4), range 5–15 (n = 34)

μg/kg • d or otherwise specified

STA treated n = 355

tachyphylaxis

n = 20 (18 %)

19.5 (±7.3), range 7.1–26.3 (n = 18)

side effects mentioned n = 111 (31 %)

GI-symptoms*

n = 23 (21 %)

17.5 (±9.5), range 6.6–27.4 (n = 20)

(transient) impairment in growth velocity

n = 15 (14 %)

17.8 (±13.5), range 7.2–55 (n = 14)

abnormal GH, IGF, IGF-BP (no growth impairment)

n = 4 (3.6 %)

25.8 (±0.72), range 25–26.3 (n = 3)

necrotizing enterocolitis

n = 8 (7 %)

21.5 (±4.7), range 15–27 (n = 6)

induration nodules/hematoma at injection site

n = 5 (4.5 %)

120 mg • month (n = 2)

decline in weight

n = 3 (2.7 %)

25 (n = 1)

asymptomatic gallstones

n = 3 (2.7 %)

33.9 (±24.8), range 6.6–55 (n = 3)

cholestatic jaundice

n = 1 (0.9 %)

40 (n = 1)

elevated liver enzymes

n = 3 (2.7 %)

16.8 (±11.4), range 10–30 (n = 3)

anaphylactic reaction

n = 1 (0.9 %)

not specified

normal growth velocity (specifically mentioned)

n = 52 (47 %)

19 (±8.7), range 8–55 (n = 32) 100 (±15.5) mg • month, range 90–120 (n = 6)

mg/kg • d or otherwise specified

GLC treated n = 15

catheter obstruction

n = 9 (60 %)

0.33 (±0.23), range 0.026–0.8 (n = 9)

side effects mentioned n = 15 (100 %)

erythema necrolyticum migrans

n = 4 (27 %)

0.3 (±0.12), range 0.22–0.46 (n = 4)

insulin autoantibodies

n = 2 (13 %)

0.2 (n = 1), 1.4 mg • d (n = 1)

subcutaneous infiltrates

n = 1 (7 %)

5 mg • d (n = 1)

no side effects (specifically mentioned)

n = 1 (7 %)

0.21 (n = 1)