Prospective follow-up of adult long-term survivors of allogeneic haematopoietic stem cell transplantation

Author(s):  
Anna Torrent ◽  
Christelle Ferrá ◽  
Montserrat Batlle ◽  
Fátima Hidalgo ◽  
Maria-José Jiménez-Lorenzo ◽  
...  
2020 ◽  
Vol 79 (8) ◽  
pp. 1084-1089
Author(s):  
Sandra van Bijnen ◽  
Jeska de Vries-Bouwstra ◽  
Cornelia H van den Ende ◽  
Maaike Boonstra ◽  
Lucie Kroft ◽  
...  

BackgroundAutologous haematopoietic stem cell transplantation (HSCT) improves survival in systemic sclerosis (SSc) with poor prognosis, but is hampered by treatment-related mortality (TRM).ObjectiveTo evaluate event-free survival (EFS), TRM, response to treatment, disease progression and patient characteristics associated with events.MethodsAll patients treated with HSCT for SSc in The Netherlands until 2017 (n=92) were included. Data on skin involvement (modified Rodnan skin score (mRSS), pulmonary function (forced vital capacity (FVC) and diffusion capacity of the lungs for carbon monoxide (DLCO)), extent of interstitial lung disease on high-resolution CT using Goh scores and left ventricular ejection fraction (LVEF) were collected at baseline, 1, 2 and 5 years. Occurrence of events, defined as death or major organ failure, were collected until 2019. As control, a comparison between patients treated with cyclophosphamide (CYC) and patients with HSCT who participated in the Autologous Stem Cell Transplantation International Scleroderma (ASTIS) trial was performed.ResultsMedian follow-up was 4.6 years. EFS estimates at 5, 10 and 15 years were 78%, 76% and 66%, respectively. Twenty deaths occurred. Mean FVC, DLCO, mRSS and Goh scores all improved significantly. Disease progression occurred in 22 patients. Frequency of TRM decreased over time and occurred more often in males. Events were independently associated with male sex, LVEF <50% and older age. In ASTIS, patients treated with HSCT (n=23) 7 events occurred versus 13 in the CYC group (n=22).ConclusionOur data confirm long-term efficacy of HSCT in improving survival, skin and lung involvement in SSc. Male sex, lower LVEF and older age at baseline were identified as risk factors for events.


Open Heart ◽  
2021 ◽  
Vol 8 (2) ◽  
pp. e001768
Author(s):  
Richard John Massey ◽  
Phoi Phoi Diep ◽  
Marta Maria Burman ◽  
Anette Borger Kvaslerud ◽  
Lorentz Brinch ◽  
...  

AimsSurvivors of allogeneic haematopoietic stem-cell transplantation (allo-HSCT) are at higher risk of cardiovascular disease. We aimed to describe right ventricular (RV) systolic function and risk factors for RV dysfunction in long-term survivors of allo-HSCT performed in their youth.Methods and resultsThis cohort included 103 survivors (53% female), aged (mean±SD) 17.6±9.5 years at allo-HSCT, with a follow-up time of 17.2±5.5 years. Anthracyclines were used as first-line therapy for 44.7% of the survivors. The RV was evaluated with echocardiography, and found survivors to have reduced RV function in comparison to a group of healthy control subjects: Tricuspid annular plane systolic excursion, (TAPSE, 20.8±3.7 mm vs 24.6±3.8 mm, p<0.001), RV peak systolic velocity (RV-s’, 11.2±2.3 cm/s vs 12.3±2.3 cm/s, p=0.001), fractional area change (FAC, 41.0±5.2% vs 42.2±5.1%, p=0.047) and RV free-wall strain (RVFWS, −27.1±4.2% vs −28.5±3.3%, p=0.043). RV systolic dysfunction (RVSD) was diagnosed in 14 (13.6%), and was strongly associated with progressive left ventricular systolic dysfunction (LVSD). High dosages of anthracyclines were associated with greater reductions in RV and LV function. Multivariable linear regressions confirmed global longitudinal strain to be a significant independent predictor for reduced RV function.ConclusionImpaired RV function was found in long-term survivors of allo-HSCT who were treated in their youth. This was associated with progressive left ventricle dysfunction, and pretransplant therapies with anthracyclines. The occurrence of RVSD was less frequent and was milder than coexisting LVSD in this cohort.


2019 ◽  
Vol 2019 ◽  
pp. 1-4
Author(s):  
Federico Meconi ◽  
Giulia Ciangola ◽  
Benedetta Mariotti ◽  
Raffaella Cerretti ◽  
Laura Cudillo ◽  
...  

Neurocysticercosis, an infection of the central nervous system with the larval stage of the cestode Taenia solium, is uncommon in developed countries. We report a case of allogeneic haematopoietic stem cell transplantation from a haploidentical donor complicated, in the long term, by T. solium infection of the central nervous system and successfully treated with empiric antiparasitic therapy with albendazole plus dexamethasone. Revised diagnostic criteria proposed by Del Brutto et al. were used for the definitive diagnosis of cerebellar neurocysticercosis.


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