Page 124 - 2021_07-Haematologica-web

Q. Zhang et al.
Assessment of treatment
Efficacy was evaluated 7, 14 and 28 days after initiating RUX therapy. The assessment of treatment was mainly based on the
Table 1. The main clinical features of enrolled patients at diagnosis.
criterion previously described in studies for pediatric HLH.8, 21 A complete response was defined as normalization of all of the quantifiable symptoms and laboratory markers of HLH, including
Clinical features
Age(years) /body weight (kg)
Sex
Duration before diagnosis Previous treatment
Patient 1*
13.2 / 41.5
Female
11 days
IVIG ganciclovir corticosteroid (10 mg/qd, 2 days)
Patient 2+
11.2 / 35.2
Male 6 days
antibiotics
Patient 3+
2.1 / 12.4
Female 4 days
–
Patient 4
1.3 / 9.1
Male 1.5 months
IVIG
Patient 5*
5.6 / 17.5
Female 21 days
IVIG ganciclovir antibiotics
Patient 6*
7.3 / 23.4
Male
13 days
ganciclovir
antibiotics corticosteroid (10 mg/qd, 3 days)
Aetiology
Fever (>38.5 °C)
Splenomegaly (size, under ribs)
Hemophagocytosis in bone marrow
Hemoglobin (g/L)
White blood cell count (x109/L)
Absolute neutrophil count (x109/L)
Absolute platelet count (x109/L)
Triglyceride (mmol/L)
Fibrinogen (g/L)
Ferritin concentration (mg/L)
Soluble CD25(pg/mL)
Natural killer cell activity (%)
Natural killer cell CD107a (%)
CNSinvolvement – – – – – –
EBV-HLH
Yes Yes
EBV-HLH
EBV-HLH Yes Yes (2 cm) Yes
78 2.87 0.76
41 5.14 2.33 1547 41075 16.04 11.20
EBV-HLH Yes
Yes (4.4 cm) Yes
93
2.9
0.63
89
4.56 1.06 3196 33950 17.02 15.25
EBV-HLH Yes Yes (3 cm) Yes 101 2.58 0.59 111 6.59 1.08 29350 71345 16.79 24.54
antibiotics AID-HLH Yes No Yes
72 2.94 0.37
19 3.31 1.05 837 19326 15.32 22.79
cm)
1039.8 22097 17.20 18.61
Patient 7 Patient 8§
2.9 / 12.5 13.4 / 40.8
Male Female 12 days 3 months
IVIG IVIG antibiotics antibiotics ganciclovir
corticosteroid corticosteroid
(5mg/qd, 4 days) (dose unclear) Unclear EBV-HLH
Yes (5 cm) Yes (3.9 Yes Yes 103 92 1.13 1.99 0.45 0.39
60 88 4.25 2.47 1.23 2.97
4375 36982 14.42 ↓ 5.44 ↓
Patient 9* Patient 10
3.3 / 13.6 2.5 / 11.2
Male Male 7 days 21 days
antibiotics antibiotics corticosteroid
(5mg/qd, 4 days)
Etiology
Fever (38.5°C)
Splenomegaly (size, under ribs)
Hemophagocytosis in bone marrow
Hemoglobin (g/L)
White blood cell count (x109/L)
Absolute neutrophil count (x109/L)
Absolute platelet count (x109/L)
Triglyceride (mmol/L)
Fibrinogen (g/L)
Ferritin concentration (mg/L)
Soluble CD25(pg/mL)
Natural killer cell activity (%)
Natural killer cell CD107a (%)
CNSinvolvement – – – – – –
IVIG: intravenous immunoglobulin; EBV-HLH: Epstein-Barr virus-associated HLH; AID: autoinflammatory disorders; CNS: central nervous system; qd: once daily. Normal reference range: Ferritin concentration< 500 mg/L, soluble CD25≤6,400pg/mL, Natural killer cell activity ≥ 15.11%, Natural killer cell CD107a ≥ 10%. #Patient diagnosed as EBV-HLH in July 2017, treated by HLH-1994 protocol and had been off all therapy for > 2 years in good health before this diagnosis. +Patients who experienced the first exposure to EBV; *Patients who were undergoing EBV reactivation;§Patient who was identified as CAEBV.
Clinical features
Age(years)/body weight (kg)
Sex
Duration before diagnosis
Previous therapy
Patient 11*
4.7 / 16.8
Male 7 days
antibiotics
Patient 12#
3.9 / 16.6
Male 7 days
antibiotics
Yes Yes No Yes (2.7 Yes Yes 99 73 1 1.5 0.29 0.49 38 42 6.2 2.69 1.7 2.57 946 3148 14219 42885 28.34 17.88 11.67 22.53
EBV-HLH Unclear Yes Yes
EBV-HLH Yes
Yes (2.8 cm) Yes
52
2.45 0.32
109
2.75
1.3 4419 44000 23.83 15.32
AIDs-HLH Yes No Yes 113 13.6
11 155 1.7 7.09 2525 16978
cm)
Yes (3.5 cm) Yes (3.6 Yes Yes 101 76 4.13 2.29 1.41 1.27
cm)
39 112 4.68 3.52 0.8 3.61 139139 4505 218875 23132 16.20 16.66 19.08 13.51
11.72 ↓ 12.26
1894
haematologica | 2021; 106(7)