Autologous Haematopoietic Stem Cell Transplantation and Systemic Sclerosis: Focus on Interstitial Lung Disease.

Gianluca Bagnato,William Neal Roberts,Egidio Imbalzano,Marianna Chiappalone,Carmelo Ioppolo,Alessandro Allegra,Natasha Irrera,Alberta De Gaetano,Giovanni Pioggia,Antonio Giovanni Versace,Daniela La Rosa,Alessandra Bitto,Sebastiano Gangemi

doi:10.3390/cells11050843

Abstract

Autologous hematopoietic stem cells transplantation (AHSCT) has been employed as treatment for severe systemic sclerosis (SSc) with high risk of organ failure. In the last 25 years overall survival and treatment-related mortality have improved, in accordance with a better patient selection and mobilization and conditioning protocols. This review analyzes the evidence from the last 5 years for AHSCT-treated SSc patients, considering in particular the outcomes related to interstitial lung disease. There are increasing data supporting the use of AHSCT in selected patients with rapidly progressive SSc. However, some unmet needs remain, such as an accurate patient selection, pre-transplantation analysis to identify subclinical conditions precluding the transplantation, and the alternatives for post-transplant ILD recurrence.

Highlights

Academic Editor: AnnaSystemic sclerosis (SSc) is a heterogeneous, systemic autoimmune disease characterized by small vessel vasculopathy, autoantibodies production, and fibroblast activation leading to fibrosis of the skin and internal organs [1]
The aim of the present review is to analyze the evidence from observational and retrospective studies and randomized clinical trials (RCTs) regarding autologous hematopoietic stem cells transplantation (AHSCT) in systemic sclerosis (SSc)-interstitial lung disease (ILD) published between January 2016 and December 2021
This study show that 90% of patients treated with hematopoietic stem cell transplantation (HSCT) or CYC present stable or reduced ILD extent assessed by HRCT, no definitive conclusion can be drawn for the superior efficacy of one regimen upon the other, possibly due to the relative short period of observation [41]

Summary

Introduction

Systemic sclerosis (SSc) is a heterogeneous, systemic autoimmune disease characterized by small vessel vasculopathy, autoantibodies production, and fibroblast activation leading to fibrosis of the skin and internal organs [1]. The clinical manifestations and the prognosis of SSc are variable: the majority of patients have skin thickening and variable involvement of internal organs. SSc-associated interstitial lung disease (SSc-ILD) represents the leading cause of mortality in patients with SSc with 3-year survival of only 52% [5]. The clinical course of SSc-ILD is variable: some patients show stable or improving forced vital capacity (FVC) while others show a progressive decline in lung function [6], that occurs more rapidly within the first few years after diagnosis and in some cases slows down. The clinical heterogeneity of the disease in association with the poor survival rate makes clinical trial enrichment difficult and complicates stratification and therapeutic management [7]

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