Allogeneic hematopoietic stem cell transplantation for severe, refractory juvenile idiopathic arthritis

Silva, Juliana M.F.; Ladomenou, Fani; Carpenter, Ben; Chandra, Sharat; Sedláček, Petr; Formánková, Renata; Grandage, Vicky; Friswell, Mark; Cant, Andrew J.; Nademi, Zohreh; Slatter, Mary; Gennery, Andrew R.; Hambleton, Sophie; Flood, Terence; Lucchini, Giovanna; Chiesa, Robert; Rao, Kanchan; Amrolia, Persis; Brogan, Paul A.; Wedderburn, Lucy R.; Glanville, Julie; Hough, Rachael; Marsh, Rebecca; Abinun, Mario; Veys, Paul

doi:10.1182/bloodadvances.2017014449

Cited by 39 publications

(37 citation statements)

References 46 publications

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“…For this purpose, it has been associated to a high risk of Chlamydia pneumoniae -associated pneumonitis in patients with chronic diseases and severe primary pulmonary hypertension in patients with scleroderma and nephritis associated to hyper-leukocytosis disease [ 36 , 37 , 38 ]. Similarly, B*35 expression has also been associated to high-risk juvenile idiopathic arthritis [ 39 ].…”

Section: Discussionmentioning

confidence: 99%

HLA Expression Correlates to the Risk of Immune Checkpoint Inhibitor-Induced Pneumonitis

Correale

Saladino

Giannarelli

et al. 2020

Cells

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Tumor-infiltrating T cell rescue by programmed cell death receptor-1 (PD-1)/PD-1 ligand-1 (PD-L1) immune checkpoint blockade is a recommended treatment for malignant diseases, including metastatic non-small-cell lung cancer (mNSCLC), malignant melanoma (MM), head and neck, kidney, and urothelial cancer. Monoclonal antibodies (mAbs) against either PD-1 or PD-L1 are active agents for these patients; however, their use may be complicated by unpredictable immune-related adverse events (irAEs), including immune-related pneumonitis (IRP). We carried out a retrospective multi-institutional statistical analysis to investigate clinical and biological parameters correlated with IRP rate on a cohort of 256 patients who received real-world treatment with PD-1/PD-L1 blocking mAbs. An independent radiological review board detected IRP in 29 patients. We did not find statistical IRP rate correlation with gender, tumor type, specific PD-1 or PD-L1 blocking mAbs, radiation therapy, inflammatory profile, or different irAEs. A higher IRP risk was detected only in mNSCLC patients who received metronomic chemotherapy +/− bevacizumab compared with other treatments prior PD-1/PD-L1 blockade. Moreover, we detected a strong correlation among the IRP rate and germinal expression of HLA-B*35 and DRB1*11, alleles associated to autoimmune diseases. Our findings may have relevant implications in predicting the IRP rate in mNSCLC patients receiving PD-1/PD-L1 blockade and need to be validated on a larger patient series.

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Section: Discussionmentioning

confidence: 99%

HLA Expression Correlates to the Risk of Immune Checkpoint Inhibitor-Induced Pneumonitis

Correale

Saladino

Giannarelli

et al. 2020

Cells

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“…Some difficult-to-treat patients deserve more experimental approaches, such as thalidomide, which requires special attention regarding the risks for thrombosis, peripheral neuropathy and teratogenicity [125, 126] or Janus kinase inhibitors [127]. In a few cases, intensive immunosuppression followed by either autologous [128] or, to minimize the risk of relapse, allogeneic haematopoietic stem cell transplantation may be considered as a last resort to control the disease [129].…”

Section: Remaining Challengesmentioning

confidence: 99%

Anakinra in children and adults with Still’s disease

et al. 2019

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Systemic juvenile idiopathic arthritis and adult-onset Still’s disease are rare autoinflammatory disorders with common features, supporting the recognition of these being one disease—Still’s disease—with different ages of onset. Anakinra was recently approved by the European Medicines Agency for Still’s disease. In this review we discuss the reasoning for considering Still’s disease as one disease and present anakinra efficacy and safety based on the available literature. The analysis of 27 studies showed that response to anakinra in Still’s disease was remarkable, with clinically inactive disease or the equivalent reported for 23–100% of patients. Glucocorticoid reduction and/or stoppage was reported universally across the studies. In studies on paediatric patients where anakinra was used early or as first-line treatment, clinically inactive disease and successful anakinra tapering/stopping occurred in >50% of patients. Overall, current data support targeted therapy with anakinra in Still’s disease since it improves clinical outcome, especially if initiated early in the disease course.

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“…Some patients clearly benefited from this treatment (31), despite considerable toxicity and non-relapse mortality (NRM) reported in the past (9, 18). The rationale for the use of allogeneic HSCT has gained additional traction more recently, as monogenic defects associated with autoimmunity and immunodeficiency have been identified (32–37).…”

Section: Introductionmentioning

confidence: 99%

Allogeneic HSCT for Autoimmune Diseases: A Retrospective Study From the EBMT ADWP, IEWP, and PDWP Working Parties

et al. 2019

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Background: This retrospective study assessed the use and long-term outcome of allogeneic hematopoietic stem cell transplantation (HSCT) in patients with severe autoimmune diseases (ADs), reported to the European Society for Blood and Marrow Transplantation (EBMT) registry. Methods: Between 1997 and 2014, 128 patients received allogeneic HSCT for various hematological ( n = 49) and non-hematological ( n = 79) refractory ADs. The median age was 12.7 years (0.2–62.2). Donors were syngeneic for seven, matched related for 46, unrelated for 51, haploidentical for 15, and cord blood for nine patients. Results: The incidence of grades II-IV acute graft-vs.-host disease (GvHD) was 20.8% at 100 days. Cumulative incidence of chronic GvHD was 27.8% at 5-years. Non-relapse mortality (NRM) was 12.7% at 100-days. Overall survival (OS) and Progression-Free Survival (PFS) were 70.2 and 59.4% at 5-years, respectively. By multivariate analysis, age <18 years, males, and more recent year of transplant were found to be significantly associated with improved PFS. Reduced conditioning intensity was associated with a lower NRM. On a subgroup of 64 patients with detailed information a complete clinical response was obtained in 67% of patients at 1-year. Conclusions: This large EBMT survey suggests the potential of allogeneic HSCT to induce long-term disease control in a large proportion of refractory ADs, with acceptable toxicities and NRM, especially in younger patients.

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Allogeneic hematopoietic stem cell transplantation for severe, refractory juvenile idiopathic arthritis

Abstract: Key Points Allo-HSCT using reduced intensity conditioning with alemtuzumab appears to be effective and safe for patients with refractory JIA. Early allo-HSCT may prevent joint damage, reduce toxicity associated with immunosuppression, and reduce transplant-related mortality.

Cited by 39 publications

References 46 publications

HLA Expression Correlates to the Risk of Immune Checkpoint Inhibitor-Induced Pneumonitis

HLA Expression Correlates to the Risk of Immune Checkpoint Inhibitor-Induced Pneumonitis

Anakinra in children and adults with Still’s disease

Allogeneic HSCT for Autoimmune Diseases: A Retrospective Study From the EBMT ADWP, IEWP, and PDWP Working Parties

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