Improved survival outcomes with anakinra over etoposide-based therapies for the management of adults with hemophagocytic lymphohistiocytosis: a retrospective multicenter research network study

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Abstract

Background:

Hemophagocytic lymphohistiocytosis (HLH) is a rare life-threatening, hyperinflammatory syndrome for which etoposide-based regimens have historically been the standard of care. Recent reports have described positive outcomes with the utilization of ruxolitinib or anakinra although these studies are often limited to small samples.

Objectives:

We aimed to compare the efficacy of ruxolitinib, anakinra, and etoposide-based therapies for the management of HLH in adult patients.

Design:

We performed a population-based, multicenter, retrospective cohort study utilizing the TriNetX Networks database.

Methods:

Adult patients (⩾18 years) diagnosed with HLH who received first-line treatment with ruxolitinib, anakinra, or etoposide between 2008 and 2023 were analyzed. The primary endpoint was overall survival (OS) at 1 year. A 1:1 propensity-score matching analysis was utilized.

Results:

Anakinra ( p = 0.020) but not ruxolitinib ( p = 0.19) resulted in a significantly higher 1-year OS when compared with etoposide-based therapies.

Conclusions:

Anakinra is effective for the management of adult patients with HLH.

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Most cited references 20

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The JAK-STAT pathway: impact on human disease and therapeutic intervention.

John J. O’Shea, Daniella M. Schwartz, Alejandro Villarino … (2015)

The Janus kinase (JAK)-signal transducer of activators of transcription (STAT) pathway is now recognized as an evolutionarily conserved signaling pathway employed by diverse cytokines, interferons, growth factors, and related molecules. This pathway provides an elegant and remarkably straightforward mechanism whereby extracellular factors control gene expression. It thus serves as a fundamental paradigm for how cells sense environmental cues and interpret these signals to regulate cell growth and differentiation. Genetic mutations and polymorphisms are functionally relevant to a variety of human diseases, especially cancer and immune-related conditions. The clinical relevance of the pathway has been confirmed by the emergence of a new class of therapeutics that targets JAKs.

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Benefit of Anakinra in Treating Pediatric Secondary Hemophagocytic Lymphohistiocytosis

Esraa Eloseily, Peter Weiser, Courtney Crayne … (2020)

To assess the benefit of the recombinant human interleukin-1 receptor antagonist anakinra in treating pediatric patients with secondary hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) associated with rheumatic and nonrheumatic conditions.

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Treatment of hemophagocytic lymphohistiocytosis with HLH-94 immunochemotherapy and bone marrow transplantation.

Shinsaku Imashuku, D Komp, Maurizio Aricò … (2002)

Hemophagocytic lymphohistiocytosis (HLH) comprises familial (primary) hemophagocytic lymphohistiocytosis (FHL) and secondary HLH (SHLH), both clinically characterized by fever, hepatosplenomegaly, and cytopenia. FHL, an autosomal recessive disease invariably fatal when untreated, is associated with defective triggering of apoptosis and reduced cytotoxic activity, resulting in a widespread accumulation of T lymphocytes and activated macrophages. In 1994 the Histiocyte Society initiated a prospective international collaborative therapeutic study (HLH-94), aiming at improved survival. It combined chemotherapy and immunotherapy (etoposide, corticosteroids, cyclosporin A, and, in selected patients, intrathecal methotrexate), followed by bone marrow transplantation (BMT) in persistent, recurring, and/or familial disease. Between July 1, 1994, and June 30, 1998, 113 eligible patients aged no more than 15 years from 21 countries started HLH-94. All had either an affected sibling (n = 25) and/or fulfilled the Histiocyte Society diagnostic criteria. At a median follow-up of 3.1 years, the estimated 3-year probability of survival overall was 55% (95% confidence interval +/- 9%), and in the familial cases, 51% (+/- 20%). Twenty enrolled children were alive and off therapy for more than 12 months without BMT. For patients who received transplants (n = 65), died prior to BMT (n = 25), or were still on therapy (n = 3), the 3-year survival was 45% (+/- 10%). The 3-year probability of survival after BMT was 62% (+/- 12%). HLH-94 is very effective, allowing BMT in most patients. Survival of children with HLH has been greatly improved.

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Author and article information

Contributors

Benjamin J. Lee:

ORCID: https://orcid.org/0000-0002-1678-1345

Role: ConceptualizationRole: Data curationRole: Formal analysisRole: InvestigationRole: MethodologyRole: Writing – original draftRole: Writing – review & editing

Journal

Journal ID (nlm-ta): Ther Adv Hematol

Journal ID (iso-abbrev): Ther Adv Hematol

Journal ID (publisher-id): TAH

Journal ID (hwp): sptah

Title: Therapeutic Advances in Hematology

Publisher: SAGE Publications (Sage UK: London, England )

ISSN (Print): 2040-6207

ISSN (Electronic): 2040-6215

Publication date (Electronic): 16 April 2024

Publication date Collection: 2024

Volume: 15

Electronic Location Identifier: 20406207241245517

Affiliations

[1-20406207241245517]Department of Pharmacy, Chao Family Comprehensive Cancer Center, University of California Irvine Health, 101 The City Drive South, Building 23, Room 275, Orange, CA 92868, USA

[2-20406207241245517]Department of Clinical Pharmacy Practice, School of Pharmacy and Pharmaceutical Sciences, University of California, Irvine, Irvine, CA, USA

Author notes

[*] benjaml9@ 123456hs.uci.edu

Author information

Benjamin J. Lee https://orcid.org/0000-0002-1678-1345

Article

Publisher ID: 10.1177_20406207241245517

DOI: 10.1177/20406207241245517

PMC ID: 11022673

PubMed ID: 38633898

SO-VID: e31ac983-d15d-4743-acd8-089b2328a73b

License:

This article is distributed under the terms of the Creative Commons Attribution 4.0 License ( https://creativecommons.org/licenses/by/4.0/) which permits any use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access pages ( https://us.sagepub.com/en-us/nam/open-access-at-sage).

History

Date received : 2 January 2024

Date accepted : 20 March 2024

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cover-date January-December 2024

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ScienceOpen disciplines: Hematology

Keywords: anakinra,etoposide,hemophagocytic lymphohistiocytosis,ruxolitinib

Data availability:

ScienceOpen disciplines: Hematology

Keywords: anakinra, etoposide, hemophagocytic lymphohistiocytosis, ruxolitinib

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Improved survival outcomes with anakinra over etoposide-based therapies for the management of adults with hemophagocytic lymphohistiocytosis: a retrospective multicenter research network study

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Abstract

Background:

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Methods:

Results:

Conclusions:

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Most cited references 20

The JAK-STAT pathway: impact on human disease and therapeutic intervention.

Benefit of Anakinra in Treating Pediatric Secondary Hemophagocytic Lymphohistiocytosis

Treatment of hemophagocytic lymphohistiocytosis with HLH-94 immunochemotherapy and bone marrow transplantation.

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