Emapalumab Treatment in Patients With Rheumatologic Disease-Associated Hemophagocytic Lymphohistiocytosis in the United States: A Retrospective Medical Chart Review Study

被引：0

作者：

Chandrakasan, Shanmuganathan ^{[1
]}

Allen, Carl E. ^{[2
]}

Bhatla, Deepika ^{[3
]}

Carter, John ^{[4
]}

Chien, May ^{[5
]}

Cooper, Robert ^{[6
]}

Draper, Lauren ^{[3
]}

Eckstein, Olive S. ^{[2
]}

Hanna, Rabi ^{[7
]}

Hays, J. Allyson ^{[8
]}

Hermiston, Michelle L. ^{[9
]}

Hinson, Ashley P. ^{[10
]}

Hobday, Patricia M. ^{[11
]}

Isakoff, Michael S. ^{[12
]}

Jordan, Michael B. ^{[13
,14
]}

Leiding, Jennifer W. ^{[15
,16
]}

Modica, Renee ^{[17
]}

Nakano, Taizo A. ^{[18
]}

Oladapo, Abiola ^{[19
,20
]}

Patel, Sachit A. ^{[21
]}

Pednekar, Priti ^{[22
,23
]}

Riskalla, Mona ^{[11
]}

Sarangi, Susmita N. ^{[24
]}

Satwani, Prakash ^{[25
]}

Tandra, Anand ^{[26
]}

Walkovich, Kelly J. ^{[27
]}

Yee, John D. ^{[20
]}

Zoref-Lorenz, Adi ^{[13
,28
]}

Behrens, Edward M. ^{[29
]}

REAL HLH investigators

机构：

[1] Emory Univ, Aflac Canc & Blood Disorders Ctr, Childrens Healthcare Atlanta, Atlanta, GA USA

[2] Baylor Coll Med, Houston, TX USA

[3] St Louis Univ, St Louis, MO USA

[4] Oregon Hlth & Sci Univ, Portland, OR USA

[5] Stanford Univ, Lucile Packard Childrens Hosp, Palo Alto, CA USA

[6] Bellflower Med Ctr, Bellflower, CA USA

[7] Cleveland Clin, Childrens Hosp, Cleveland, OH USA

[8] Childrens Mercy Hosp, Kansas City, MO USA

[9] Univ Calif San Francisco, San Francisco, CA USA

[10] Atrium Hlth, Charlotte, NC USA

[11] Univ Minnesota, Med Sch, Minneapolis, MN USA

[12] Connecticut Childrens Med Ctr, Hartford, CT USA

[13] Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH USA

[14] Univ Cincinnati, Coll Med, Cincinnati, OH USA

[15] Johns Hopkins Univ, Baltimore, MD USA

[16] Johns Hopkins All Childrens Hosp, St Petersburg, FL USA

[17] Univ Florida Hlth, Shands Childrens Hosp, Gainesville, FL USA

[18] Childrens Hosp Colorado, Aurora, CO USA

[19] Apnimed, Cambridge, MA USA

[20] Sobi Inc, Waltham, MA USA

[21] Univ Nebraska Med Ctr, Omaha, NE USA

[22] Astellas Pharma Inc, Northbrook, IL USA

[23] PRECISIONheor, Bethesda, MD USA

[24] Medstar Georgetown Univ Hosp, Washington, DC USA

[25] NewYork Presbyterian Columbia Univ, Irving Med Ctr, New York, NY USA

[26] Franciscan Hlth, Indianapolis, IN USA

[27] Univ Michigan, Med Sch, Ann Arbor, MI USA

[28] Tel Aviv Med Ctr & Sch Med, Dept Pediat Surg, Tel Aviv, Israel

[29] Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA

来源：

ARTHRITIS & RHEUMATOLOGY | 2025年 / 77卷 / 02期

关键词：

MACROPHAGE ACTIVATION SYNDROME; JUVENILE IDIOPATHIC ARTHRITIS; INTERFERON-GAMMA; PATHOPHYSIOLOGY; INTERLEUKIN-18; TOCILIZUMAB; CHILDREN; THERAPY; ADULTS;

D O I：

暂无

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

ObjectiveRheumatologic disease-associated hemophagocytic lymphohistiocytosis (HLH), a rare, life-threatening, systemic hyperinflammatory syndrome, occurs as a complication of underlying rheumatologic disease. Real-world evidence is lacking on emapalumab, a fully human monoclonal antibody that neutralizes the proinflammatory cytokine interferon-gamma, approved for treating patients with primary HLH.MethodsREAL-HLH, a retrospective medical chart review study conducted across 33 US hospitals, assessed real-world treatment patterns and outcomes in patients with HLH treated with one or more dose of emapalumab between November 20, 2018, and October 31, 2021. Data are presented for the subset of patients with rheumatologic disease-associated HLH.ResultsFifteen of 105 patients (14.3%) had rheumatologic disease-associated HLH. Of these, nine (60.0%) had systemic juvenile idiopathic arthritis, and one (6.7%) had adult-onset Still disease. Median (range) age at HLH diagnosis was 5 (0.9-39) years. Most patients (9 of 15; 60.0%) initiated emapalumab in an intensive care unit. Emapalumab was most frequently initiated for treating refractory or recurrent (10 of 15; 66.7%) disease. Most patients received HLH-related therapies before (10 of 15; 66.7%) and concurrently with (15 of 15; 100.0%) emapalumab. Emapalumab-containing regimens stabilized or achieved physician-determined normalization of most laboratory parameters, including absolute neutrophil count and absolute lymphocyte count (13 of 14; 92.9%), chemokine ligand 9 (9 of 11; 81.8%), and platelets and alanine transaminase (11 of 14; 78.6%), and reduced glucocorticoid dose by 80%. Overall survival and 12-month survival probability from emapalumab initiation were 86.7%.ConclusionEmapalumab-containing regimens stabilized or normalized most key laboratory parameters, reduced glucocorticoid dose, and were associated with low disease-related mortality, thereby demonstrating potential benefits in patients with rheumatologic disease-associated HLH.

引用

页码：226 / 238

页数：13

共 45 条

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