Effect of COVID‐19 on anakinra‐induced remission in homozygous STX11 hemophagocytosis lymphohistiocytosis

• Published in: Pediatric Blood & Cancer Vol. 68; no. 6; pp. e28897 - n/a
• Main Authors: Vagrecha, Anshul, Patel, Hiren B., Mamdouhi, Tania, Acharya, Suchitra, Appiah‐Kubi, Abena, Aygun, Banu, Vlachos, Adrianna, Wolfe, Lawrence C., Lipton, Jeffrey M., Cron, Randy Q., Fein Levy, Carolyn
• Format: Journal Article Web Resource
• Language: English
• Published: United States John Wiley & Sons, Inc 01.06.2021; Wiley Subscription Services, Inc; John Wiley and Sons Inc
• Subjects: Anti-Inflammatory Agents - administration & dosage; Anti-Inflammatory Agents - therapeutic use; Child, Preschool; COVID-19; COVID-19 - complications; COVID-19 - drug therapy; Frameshift Mutation; Hematology; Homozygote; Humans; Interleukin 1 receptor antagonist; Interleukin 1 Receptor Antagonist Protein - administration & dosage; Interleukin 1 Receptor Antagonist Protein - therapeutic use; Letter to the Editor; Letters to the Editor; Lymphohistiocytosis, Hemophagocytic - complications; Lymphohistiocytosis, Hemophagocytic - drug therapy; Lymphohistiocytosis, Hemophagocytic - genetics; Male; Oncology; Pediatrics; Qa-SNARE Proteins - genetics; Remission; SARS-CoV-2 - isolation & purification
• ISSN: 1545-5009; 1545-5017
• DOI: 10.1002/pbc.28897

To the Editor: Primary hemophagocytic lymphohistiocytosis (pHLH) is a frequently fatal disease characterized by excessive cytokine‐mediated immune cell activation associated with homozygous mutations of genes encoding proteins important for perforin‐mediated cytolysis (PRF1, UNC13D, STX11, RAB27A, and STXBP2).1 Recommended treatment, based on the HLH‐2004 trial, consists of chemotherapy with dexamethasone and etoposide followed by hematopoietic stem cell transplant (HSCT). Anticytokine therapy to treat pHLH such as the recently FDA‐approved anti‐interferon‐gamma antibody emapalumab3 or the previously reported interleukin 1 (IL‐1) receptor antagonist anakinra4-6 appear to be associated with decreased morbidity and mortality. In the treatment of pHLH, the recent shift toward the use of biologic agents offers promise in disease control with minimal morbidity.3 While anakinra has shown to be effective in the management of sHLH, malignancy‐associated HLH,4, 5, 13 and macrophage activation syndrome (MAS),14, 15 there is only one prior report on its use in pHLH as part of a multiagent regimen.6 Herein, we highlight the successful management of pHLH using anakinra monotherapy. Tofacitinib for sJIA Adjudication of MAS Committee Chair, Pfizer, 2017‐present; strategies for transitioning one therapy to another, Consultant, Novartis, Houston, TX, 2019; changing treatment landscape for MAS/sHLH advisory board, SOBI, Atlanta, GA, 2019; “The Cytokine Storm Syndrome: What clinicians need to know” WebMD funded by Sobi, 2020; “A Tale of Two Storms: COVID‐19 and the Cytokine Storm” WebMD funded by Sobi 2020; “COVID‐19 from Cytokine Signature to Cytokine Storm” WebMD funded by Sobi 2020; Co‐Principal Investigator on an ongoing investigator‐initiated clinical trial to study anakinra in a randomized, double‐blinded placebo‐controlled trial for treating hemophagocytic lymphohistiocytosis in children and adults (funded by SOBI).