Dataset Information

Long-term follow-up after lymphodepleting autologous haematopoietic cell transplantation for treatment-resistant systemic lupus erythematosus.

ABSTRACT:

Objective

Autologous haematopoietic cell transplantation (AHSCT) improves immunologic dysfunction in patients with SLE. However, the curative potential of this therapy remains uncertain. This study reports outcomes in SLE patients receiving a lymphodepleting, reduced intensity regimen for AHSCT in SLE.

Methods

Eight patients with SLE refractory to treatment, including i.v. cyclophosphamide (CYC), were enrolled. Five had LN and three CNS involvement as primary indications for transplant. Haematopoietic cell mobilization with CYC, G-CSF and rituximab was followed by collection of CD34+ positively selected cells. The conditioning regimen consisted of concurrent administration of CYC, fludarabine and rituximab. All immunosuppressive medications were discontinued at the start of mobilization and CS were rapidly tapered after the transplant.

Results

Five of eight patients achieved a complete response, including a decline in the SLEDAI to zero, which was sustained in four patients for a median of 165 months (range 138-191). One patient achieved a partial response, which was followed by relapse at month 18. Two patients with nephritis and underlying comorbidities in most organs had early deaths from infection and multiorgan failure. AHSCT resulted in profound lymphodepletion, followed by expansion of Treg cells and repopulation of naive T and B cells. Patients with a complete response showed a sustained suppression of the SLE-associated IFN-induced gene signature, marked depletion of memory and plasmablast B cells and resultant sustained elimination of anti-dsDNA antibody.

Conclusion

Durable clinical and serologic remissions with suppression in the IFN gene signature can be achieved in refractory SLE following lymphodepleting AHSCT.

Trial registration

ClinicalTrials.gov, https://clinicaltrials.gov, NCT00076752.

SUBMITTER: Goklemez S

PROVIDER: S-EPMC9348613 | biostudies-literature | 2022 Aug

REPOSITORIES: biostudies-literature

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Publications

Long-term follow-up after lymphodepleting autologous haematopoietic cell transplantation for treatment-resistant systemic lupus erythematosus.

Goklemez Sencer S Hasni Sarfaraz S Hakim Frances T FT Muraro Paolo A PA Pirsl Filip F Rose Jeremy J Memon Sarfraz S Fowler Daniel F DF Steinberg Seth M SM Baker Eva H EH Panch Sandya R SR Gress Ronald R Illei Gabor G GG Lipsky Peter E PE Pavletic Steven Z SZ

Rheumatology (Oxford, England) 20220801 8

<h4>Objective</h4>Autologous haematopoietic cell transplantation (AHSCT) improves immunologic dysfunction in patients with SLE. However, the curative potential of this therapy remains uncertain. This study reports outcomes in SLE patients receiving a lymphodepleting, reduced intensity regimen for AHSCT in SLE.<h4>Methods</h4>Eight patients with SLE refractory to treatment, including i.v. cyclophosphamide (CYC), were enrolled. Five had LN and three CNS involvement as primary indications for trans ...[more]

PMID: 34875023

Similar Datasets

Project description:IntroductionPatients with systemic lupus erythematous were vulnerable to severe coronavirus disease 2019 infection and the negative impact of disrupted healthcare delivery. Telemedicine has been a popular alternative to standard in-person care during the pandemic despite the lack of evidence.MethodsThis was a 1-year pragmatic randomized-controlled trial. Patients followed at the lupus nephritis clinic were randomized to either telemedicine or standard follow-up in a 1:1 ratio. Patients in the telemedicine group were followed up via videoconferencing. Standard follow-up group patients continued conventional in-person outpatient care. The primary outcome of the study was the proportion of patients in low disease activity after 1 year. Secondary outcomes included cost-of-illness, safety, and various patient-reported outcomes.ResultsFrom 6/2020 to 12/2021, 144 patients were randomized and 141 patients (telemedicine: 70, standard follow-up: 71) completed the study. At 1 year, 80.0% and 80.2% of the patients in the telemedicine group and standard follow-up group were in lupus low disease activity state or complete remission, respectively (p = 0.967). Systemic lupus erythematous disease activity indices, number of flares and frequency of follow-ups were also similar. There were no differences in the cost-of-illness, quality of life or mental health scores. However, significantly more patients in the telemedicine group (41.4% vs 5.6%; p < 0.001) required switch of mode of follow-up and higher proportion of them had hospitalization during the study period (32.9% vs 15.5%; p = 0.016). Being in the telemedicine group or not in low disease activity at baseline were the independent predictors of hospitalization (odds ratio: 2.6; 95% confidence interval: 1.1-6.1, odds ratio: 2.7, 95% confidence interval: 1.1-6.7, respectively) in the post hoc analysis.ConclusionsIn patients with systemic lupus erythematous, telemedicine predominant follow-up resulted in similar 1-year disease control compared to standard care. However, it needed to be complemented by in-person visits, especially in patients with unstable disease.

Dataset Information

Long-term follow-up after lymphodepleting autologous haematopoietic cell transplantation for treatment-resistant systemic lupus erythematosus.

Objective

Methods

Results

Conclusion

Trial registration

Publications

Long-term follow-up after lymphodepleting autologous haematopoietic cell transplantation for treatment-resistant systemic lupus erythematosus.

Similar Datasets

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