Allogeneic Hematopoietic Cell Transplant for Systemic Juvenile Idiopathic Arthritis and Macrophage Activation Syndrome.

Nicole Davidson,Rolla F Abu-Arja,Rajinder P S Bajwa,Hemalatha G Rangarajan,Veronika Polishchuk,Kyla Driest

doi:10.1155/2021/9323141

Abstract

Systemic juvenile idiopathic arthritis (sJIA) is characterized by arthritis, fever, rash, lymphadenopathy, hepatosplenomegaly, and serositis. Macrophage activation syndrome is the most feared complication of sJIA with a high risk of mortality. We report a 16-year-old female diagnosed with refractory systemic juvenile idiopathic arthritis (sJIA) complicated by recurrent macrophage activation syndrome (MAS), severe joint disease, and lung involvement requiring prolonged immunosuppressive therapy. She received a matched unrelated allogeneic hematopoietic cell transplant (Allo-HCT) using a reduced-intensity conditioning regimen and is now, 3 years after the transplant, with complete resolution of sJIA symptoms, off immunosuppressants, and with significant improvement in the quality of life.

Highlights

Juvenile idiopathic arthritis (JIA) is the most common childhood chronic rheumatologic disorder and used to describe several forms of arthritis that develop in children under age 16 [1]
macrophage activation syndrome (MAS) is a form of secondary hemophagocytic lymphohistiocytosis (HLH) that manifests with overproduction of cytokines and a hyperinflammatory state associated with cytopenia, liver dysfunction, and coagulopathy, resembling disseminated intravascular coagulation and, in severe cases, leading to organ failure [4, 5]. erapy includes nonsteroidal antiinflammatory drugs, long-term use of corticosteroids, calcineurin inhibitors, intravenous immunoglobulin, and other biologic agents, with anakinra being the most selected agent [6, 7]
Over a course of 3 years, she was treated with multiple immunosuppressive therapies including adalimumab, tocilizumab, prednisone, intravenous immunoglobulin, anakinra, canakinumab, leflunomide, methotrexate, cyclosporine, and tacrolimus

Summary

Introduction

Juvenile idiopathic arthritis (JIA) is the most common childhood chronic rheumatologic disorder and used to describe several forms of arthritis that develop in children under age 16 [1]. A life-threatening complication (mortality rate: 8%–17%) of sJIA is macrophage activation syndrome (MAS) [3]. We describe a female who is 22 years old with refractory sJIA and recurrent MAS who received a matched unrelated allogeneic hematopoietic cell transplant (Allo-HCT). Over a course of 3 years, she was treated with multiple immunosuppressive therapies including adalimumab, tocilizumab, prednisone, intravenous immunoglobulin, anakinra, canakinumab, leflunomide, methotrexate, cyclosporine, and tacrolimus.

Results

Conclusion