Journal Article

Case report: Success of allogeneic hematopoietic stem cell transplantation for refractory systemic-onset juvenile idiopathic arthritis

Proulx, Catherine, El-Jalbout, Ramy, Morin, Marie-Paule, Couture, Julie, Samaan, Kathryn, Barsalou, Julie, Decaluwe, Hélène, Teira, Pierre, Blincoe, Annaliesse, Bittencourt, Henrique, Cellot, Sonia, Beaufils, Camille, Kleiber, Niina, Touzot, Fabien, Haddad, Elie, De Bruycker, Jean Jacques, Duval, Michel

2023

Overview

ObjectivesThis study reports cases of systemic-onset juvenile idiopathic arthritis (sJIA) who underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT) at our center and reviews published outcomes of allo-HSCT in sJIA.MethodsWe present a case report of two patients with sJIA who underwent allo-HSCT at a tertiary pediatric hospital. Each patient’s disease course and allo-HSCT protocol/outcome are described. Outcomes of published cases of allo-HSCT in sJIA were compared to our experience.ResultsTwo patients with sJIA had allo-HSCT. Both failed multiple lines of disease-modifying anti-rheumatic drugs and experienced severe disease/treatment-related complications. Despite post-HSCT complications, both recovered without sequelae. Five years post-HSCT, patient 1 is in complete remission (CR) and is off medications. Patient 2 was in CR until 11 months post-HSCT after which he developed three disease flares. At 4 years post-HSCT he is currently in CR on Adalimumab monotherapy. Engraftment was excellent with a donor chimerism of 100% for patient 1 and 93% for patient 2. In the literature, the outcome of allo-HSCT is reported in 13 sJIA patients. When merging those with our 2 patients, 1/15 patients died and 13/14 achieved CR, of which 12 are off medications (median [range] follow-up: 2.2 [0.2–7.0] years). Extended follow-up data on 11 of the 13 reported sJIA patients showed that an additional 3 patients flared at 3, 4, and 10 years post-HSCT.ConclusionWe report two patients with severe/refractory sJIA who underwent successful allo-HSCT and achieved CR. Allo-HSCT is a potential curative option for severe/refractory sJIA. It should be considered only after failure of conventional sJIA treatments and when an HLA-matched donor is available in order to lower transplant-related mortality. The outcomes of reported sJIA patients who received allo-HSCT are encouraging but long-term follow-up data are needed to better characterized the risk–benefit ratio of this procedure.

• Publisher: Frontiers Media SA,Frontiers Media S.A
• Subject: allogeneic hematopoietic stem cell transplantation / Anti-inflammatory agents / Arthritis / Blood & organ donations / Blood pressure / Body mass index / Bone marrow / bone marrow transplantation / Case reports / Drug dosages / Epidural / Epstein-Barr virus / Fatty liver / Fever / Fractures / Genetic testing / Graft versus host disease / Habitus / juvenile idiopathic arthritis / Medicine / Mucositis / Patients / pediatric rheumatic disease / Pneumonia / Remission (Medicine) / Sleep apnea / Spinal cord / Spinal stenosis / Stem cell transplantation / Steroids / systemic-onset juvenile idiopathic arthritis