Diversity of peripheral blood human NK cells identified by single-cell RNA sequencing

SL Smith, PR Kennedy, KB Stacey… - Blood …, 2020 - ashpublications.org
SL Smith, PR Kennedy, KB Stacey, JD Worboys, A Yarwood, S Seo, EH Solloa, B Mistretta…
Blood advances, 2020ashpublications.org
Children with immune thrombocytopenia (ITP) rarely suffer from life-threatening bleeds (eg,
intracranial hemorrhage). In such settings, the combination of IV methylprednisolone (IVMP)
with IV immune globulin (IVIG) is used to rapidly increase platelet counts (PCs). However,
there are no controlled data to support using combination therapy over IVIG alone. We
conducted a randomized, double-blind, placebo-controlled study to evaluate the rapidity of
the PC increment and associated adverse events (AEs) between 2 regimens: A (IV placebo) …
Abstract
Children with immune thrombocytopenia (ITP) rarely suffer from life-threatening bleeds (eg, intracranial hemorrhage). In such settings, the combination of IV methylprednisolone (IVMP) with IV immune globulin (IVIG) is used to rapidly increase platelet counts (PCs). However, there are no controlled data to support using combination therapy over IVIG alone. We conducted a randomized, double-blind, placebo-controlled study to evaluate the rapidity of the PC increment and associated adverse events (AEs) between 2 regimens: A (IV placebo) and B (IVMP 30 mg/kg), both given over 1 hour, followed in both cases by IVIG (Gamunex 10%) 1 g/kg over 2-3 hours in children 1-17 years old with primary ITP and PCs <20 × 109/L in whom physicians had decided to treat with IVIG. Thirty-two children (ages: median, 8 years; range, 1.2-17.5 years) with a mean baseline PC of 9.2 × 109/L participated. Eighteen were randomized to regimen A and 14 to regimen B. By 8 hours after initiating therapy, 55% of all children had a PC ≥20 × 109/L (no group difference). By 24 hours, mean PCs were 76.9 × 109/L (B) vs 55 × 109/L (A) (P = .06; P = .035 when adjusted for intergroup differences in patient ages). No patient experienced severe bleeding/unexpected severe AEs. There were statistically fewer IVIG-related headaches in the group receiving combination therapy (P = .046). Our findings show a rapid response to IVIG with/without steroids and provide evidence to support the use of IVMP+IVIG in life-threatening situations. This trial was registered at www.clinicaltrials.gov as #NCT00376077.
ashpublications.org