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
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Scientific paper - Original scientific paper
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 More authors...

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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, no. 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, no. 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, and 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]. 2021 March 18 [cited 2024 April 12];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, no. 3, March 2021. [Online]. Available at: https://urn.nsk.hr/urn:nbn:hr:105:008893. [Accessed: 12 April 2024]

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Metadata
Title (english)A randomised, open-label trial to assess the optimal treatment strategy in early diffuse cutaneous systemic sclerosis: the UPSIDE study protocol
AuthorJulia Spierings
AuthorAnna van Rhenen
AuthorPaco MW Welsing
AuthorAnne CA Marijnissen
AuthorEllen De Langhe
AuthorNicoletta Del Papa
AuthorDaan Dierickx
AuthorKarina R Gheorghe
AuthorJoerg Henes
AuthorRoger Hesselstrand
AuthorTessa Kerre
AuthorPer Ljungman
AuthorArjan A van de Loosdrecht
AuthorErik WAF Marijt
AuthorMiro Mayer
AuthorMarc Schmalzing
AuthorRoland Schroers
AuthorVanessa Smith
AuthorReinhard E Voll
AuthorMadelon C Vonk
AuthorAlexandre E Voskuyl
AuthorJeska K de Vries-Bouwstra
AuthorUlrich A Walker
AuthorDirk M Wuttge
AuthorJacob M van Laar
Author's institutionUniversity of Zagreb
School of Medicine
(Department of Internal Medicine)
Scientific / art field,
discipline and subdiscipline		BIOMEDICINE AND HEALTHCARE
Clinical Medical Sciences
Internal Medicine
Abstract (english)
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.
Keywords (english)
Languageenglish
Publication typeScientific paper - Original scientific paper
Publication statusPublished
Peer reviewPeer review
Publication versionPublished version
Journal titleBMJ Open
Numberingvol. 11, no. 3, e044483
p-ISSN2044-6055
e-ISSN2044-6055
DOIhttps://doi.org/10.1136/bmjopen-2020-044483
URN:NBNurn:nbn:hr:105:008893
Publication2021-03-18
Document URLhttps://bmjopen.bmj.com/lookup/doi/10.1136/bmjopen-2020-044483
Type of resourceText
Access conditionsOpen access
Terms of useLicence Attribution-NonCommercial 4.0 International (CC BY-NC 4.0) icon
Created on2021-10-04 09:40:40