prikaz prve stranice dokumenta A randomised, open-label trial to assess the optimal treatment strategy in early diffuse cutaneous systemic sclerosis: the UPSIDE study protocol
  Preuzmi
PDF 350.09 KB
Znanstveni rad - Izvorni znanstveni rad
A randomised, open-label trial to assess the optimal treatment strategy in early diffuse cutaneous systemic sclerosis: the UPSIDE study protocol
BMJ Open, 11 (2021), 3; e044483. https://doi.org/10.1136/bmjopen-2020-044483
Spierings, Julia; van Rhenen, Anna; Welsing, Paco MW; Marijnissen, Anne CA; De Langhe, Ellen; Del Papa, Nicoletta; Dierickx, Daan; Gheorghe, Karina R; Henes, Joerg; Hesselstrand, Roger; Kerre, Tessa; Ljungman, Per; van de Loosdrecht, Arjan A; Marijt, Erik WAF; Mayer, Miro; Schmalzing, Marc; Schroers, Roland; Smith, Vanessa; Voll, Reinhard E; Vonk, Madelon C; Voskuyl, Alexandre E; de Vries-Bouwstra, Jeska K; Walker, Ulrich A; Wuttge, Dirk M; van Laar, Jacob M Više autora...

Citirajte ovaj rad

Spierings, J., van Rhenen, A., Welsing, P. M., Marijnissen, A. C., De Langhe, E., Del Papa, N. ... van Laar, J. M. (2021). A randomised, open-label trial to assess the optimal treatment strategy in early diffuse cutaneous systemic sclerosis: the UPSIDE study protocol. BMJ Open, 11. (3). doi: 10.1136/bmjopen-2020-044483

Spierings, Julia, et al. "A randomised, open-label trial to assess the optimal treatment strategy in early diffuse cutaneous systemic sclerosis: the UPSIDE study protocol." BMJ Open, vol. 11, br. 3, 2021. https://doi.org/10.1136/bmjopen-2020-044483

Spierings, Julia, Anna van Rhenen, Paco MW Welsing, Anne CA Marijnissen, Ellen De Langhe, Nicoletta Del Papa, Daan Dierickx, et al. "A randomised, open-label trial to assess the optimal treatment strategy in early diffuse cutaneous systemic sclerosis: the UPSIDE study protocol." BMJ Open 11, br. 3 (2021). https://doi.org/10.1136/bmjopen-2020-044483

Spierings, J., et al. (2021) 'A randomised, open-label trial to assess the optimal treatment strategy in early diffuse cutaneous systemic sclerosis: the UPSIDE study protocol', BMJ Open, 11(3). doi: 10.1136/bmjopen-2020-044483

Spierings J, van Rhenen A, Welsing PM, Marijnissen AC, De Langhe E, Del Papa N, i sur.. A randomised, open-label trial to assess the optimal treatment strategy in early diffuse cutaneous systemic sclerosis: the UPSIDE study protocol. BMJ Open [Internet]. 18.03.2021. [pristupljeno 05.10.2024.];11(3). doi: 10.1136/bmjopen-2020-044483

J. Spierings, et al., "A randomised, open-label trial to assess the optimal treatment strategy in early diffuse cutaneous systemic sclerosis: the UPSIDE study protocol", BMJ Open, vol. 11, br. 3, Ožujak 2021. [Online]. Dostupno na: https://urn.nsk.hr/urn:nbn:hr:105:008893. [Citirano: 05.10.2024.]

Za citiranje koristite ovu mrežnu adresu: https://urn.nsk.hr/urn:nbn:hr:105:008893
Prijavite se u repozitorij kako biste mogli spremiti objekt u svoju listu.
Podaci o radu
Naslov (engleski)A randomised, open-label trial to assess the optimal treatment strategy in early diffuse cutaneous systemic sclerosis: the UPSIDE study protocol
AutorJulia Spierings
AutorAnna van Rhenen
AutorPaco MW Welsing
AutorAnne CA Marijnissen
AutorEllen De Langhe
AutorNicoletta Del Papa
AutorDaan Dierickx
AutorKarina R Gheorghe
AutorJoerg Henes
AutorRoger Hesselstrand
AutorTessa Kerre
AutorPer Ljungman
AutorArjan A van de Loosdrecht
AutorErik WAF Marijt
AutorMiro Mayer
AutorMarc Schmalzing
AutorRoland Schroers
AutorVanessa Smith
AutorReinhard E Voll
AutorMadelon C Vonk
AutorAlexandre E Voskuyl
AutorJeska K de Vries-Bouwstra
AutorUlrich A Walker
AutorDirk M Wuttge
AutorJacob M van Laar
Autorova ustanovaSveučilište u Zagrebu
Medicinski fakultet
(KATEDRA ZA INTERNU MEDICINU)
Znanstveno / umjetničko
područje, polje i grana		BIOMEDICINA I ZDRAVSTVO
Kliničke medicinske znanosti
Interna medicina
Sažetak (engleski)
Introduction: Systemic sclerosis (SSc) is a chronic, autoimmune connective tissue disease associated with high morbidity and mortality, especially in diffuse cutaneous SSc (dcSSc). Currently, there are several treatments available in early dcSSc that aim to change the disease course, including immunosuppressive agents and autologous haematopoietic stem cell transplantation (HSCT). HSCT has been adopted in international guidelines and is offered in current clinical care. However, optimal timing and patient selection for HSCT are still unclear. In particular, it is unclear whether HSCT should be positioned as upfront therapy or rescue treatment for patients refractory to immunosuppressive therapy. We hypothesise that upfront HSCT is superior and results in lower toxicity and lower long-term medical costs. Therefore, we propose this randomised trial aiming to determine the optimal treatment strategy for early dcSSc by comparing two strategies used in standard care: (1) upfront autologous HSCT versus (2) immunosuppressive therapy (intravenous cyclophosphamide pulse therapy followed by mycophenolate mofetil) with rescue HSCT in case of treatment failure. ----- Methods and analysis: The UPSIDE (UPfront autologous hematopoietic Stem cell transplantation vs Immunosuppressive medication in early DiffusE cutaneous systemic sclerosis) study is a multicentre, randomised, open-label, controlled trial. In total, 120 patients with early dcSSc will be randomised. The primary outcome is event-free survival at 2 years after randomisation. Secondary outcomes include serious adverse events, functional status and health-related quality of life. We will also evaluate changes in nailfold capillaroscopy pattern, pulmonary function, cardiac MR and high-resolution CT of the chest. Follow-up visits will be scheduled 3-monthly for 2 years and annually in the following 3 years. ----- Ethics and dissemination: The study was approved by the Dutch Central Committee on Research Concerning Human Subjects (NL72607.041.20). The results will be disseminated through patient associations and conventional scientific channels.
Ključne riječi (engleski)
Jezikengleski
Vrsta publikacijeZnanstveni rad - Izvorni znanstveni rad
Status objaveObjavljen
Vrsta recenzijeRecenziran
Verzija publikacijeObjavljena verzija rada (izdavačev PDF)
Naslov časopisaBMJ Open
Brojčani podacivol. 11, br. 3, e044483
p-ISSN2044-6055
e-ISSN2044-6055
DOIhttps://doi.org/10.1136/bmjopen-2020-044483
URN:NBNurn:nbn:hr:105:008893
Datum objave publikacije2021-03-18
URL dokumentahttps://bmjopen.bmj.com/lookup/doi/10.1136/bmjopen-2020-044483
Vrsta resursaTekst
Prava pristupaOtvoreni pristup
Uvjeti korištenjaIkona licence Imenovanje-Nekomercijalno 4.0 međunarodna (CC BY-NC 4.0)
Datum i vrijeme pohrane2021-10-04 09:40:40