scholarly journals Information preferences about treatment options in diffuse cutaneous systemic sclerosis: A Delphi consensus study

2021 ◽  
pp. 239719832110433
Author(s):  
Julia Spierings ◽  
Hilde Nienhuis ◽  
Eva van Lieshout ◽  
Jacob M van Laar ◽  
Arwen H Pieterse
Keyword(s):  
Decision Making ◽  
Stem Cell ◽  
Mycophenolate Mofetil ◽  
Systemic Sclerosis ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Treatment Options ◽  
Information Preferences ◽  
The Third ◽  
Diffuse Cutaneous Systemic Sclerosis

Objectives: The aim of this study was to identify and prioritize aspects essential for decision making in patients with diffuse cutaneous systemic sclerosis (dcSSc) and to gain insight into information preferences of treatment options which could guide development of a leaflet for patients. Methods: A three-round Delphi study was conducted with a panel of patients with dcSSc. The questionnaire was based on a systematic literature search regarding benefits and harms of four main treatment options in dcSSc: methotrexate, mycophenolate mofetil, cyclophosphamide pulses and stem cell transplantation. Patients were asked to identify information that is essential for making a treatment decision. After the third round, a live, online discussion was held in order to reach consensus on these items and to discuss the content and design of the leaflet. Consensus was defined as ⩾75% agreement among panel members. Results: Of the 36 patients invited, 78% (n = 28) participated in one or more rounds, 67% (n = 24) completed the first, 69% (n = 25) the second and 75% (n = 27) the third round. In the last round, median age of participants was 51 years (interquartile range, 18) and median disease duration 4 years (interquartile range, 5); 52% were female. Patients had been treated with mycophenolate mofetil (67%), methotrexate (44%), cyclophosphamide (41%), autologous stem cell transplantation (26%), rituximab (4%) or were treatment-naïve (7%). Eight patients joined the live panel discussion. The panel reached consensus on seven benefits (prolonged progression-free survival, improved quality of life, improved daily functioning, improved pulmonary function, improved skin thickness, improved mobility and reduced fatigue) and four harms (treatment-related mortality, infections, cardiac damage, increased risk of cancer) as essential information for decision making. Also a design of a leaflet was made. Conclusion: This study identified information about treatment options in dcSSc that should be addressed with patients. Our results can be used to develop effective patient information.

scholarly journals From “being at war” to “getting back on your feet”: A qualitative study on experiences of patients with systemic sclerosis treated with hematopoietic stem cell transplantation

2020 ◽  
Vol 5 (3) ◽  
pp. 202-209
Author(s):  
Julia Spierings ◽  
Carolijn JM de Bresser ◽  
Femke CC van Rhijn-Brouwer ◽  
Arwen Pieterse ◽  
Madelon C Vonk ◽  
...  
Keyword(s):  
Stem Cell ◽  
Systemic Sclerosis ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Social Issues ◽  
Well Being ◽  
Hematopoietic Stem ◽  
Diffuse Cutaneous Systemic Sclerosis

Objectives: To gain insight into the experiences of patients with diffuse cutaneous systemic sclerosis during and after autologous hematopoietic stem cell transplantation. Methods: Semi-structured interviews were conducted with patients who underwent hematopoietic stem cell transplantation in four university hospitals in the Netherlands. Interviews were transcribed verbatim and thematically analyzed. Results: Nine male and seven female patients were interviewed, median age 47 years (range: 27–68). Patients mentioned their life was severely disrupted before hematopoietic stem cell transplantation and remained unsettled a long time after treatment. Uncertainty because of disease progression, loss of control over health and the sense of time and fear of treatment-related adverse events were common during hospitalization. After hematopoietic stem cell transplantation, patients experienced more physical limitations than they had expected, and recovery took longer and was mentally taxing. Going back to work and finding a new balance in personal relations and social life was complicated. Patients described various strategies to deal with challenges. Family and friends provided essential support, although many experienced a dwindling social circle. Most patients also appreciated peer support. All patients were satisfied with the low threshold for contact with physicians and nurses during hospitalization. However, aftercare focused on medical aspects rather than on psychological well-being and social issues. Moreover, patients would have preferred to be better prepared on what to expect after discharge, and lacked information about self-management, prognosis, optimal recovery, work, sexuality, and family planning. Conclusion: Hematopoietic stem cell transplantation has a major physical and psychological impact on patients with diffuse cutaneous systemic sclerosis. The course of recovery after this intensive therapy was unexpectedly long for some patients and offer of support was far less pro-active post-HSCT compared to pre-HSCT and during HSCT.

scholarly journals Rituximab as a Treatment Option after Autologous Hematopoietic Stem Cell Transplantation in a Patient with Systemic Sclerosis

2021 ◽  
Vol 11 (7) ◽  
pp. 600
Author(s):  
Paul Gressenberger ◽  
Philipp Jud ◽  
Gabor Kovacs ◽  
Sonja Kreuzer ◽  
Hans-Peter Brezinsek ◽  
...  
Keyword(s):  
Stem Cell ◽  
Systemic Sclerosis ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Treatment Option ◽  
Clinical Symptoms ◽  
Stable Condition ◽  
Hematopoietic Stem

Systemic sclerosis (SSc) is an intractable autoimmune disease characterized by vasculopathy and organ fibrosis. Autologous hematopoietic stem cell transplantation (AHSCT) should be considered for the treatment of selected patients with rapid progressive SSc at high risk of organ failure. It, however, remains elusive whether immunosuppressive therapies such as rituximab (RTX) are still necessary for such patients after AHSCT, especially in those with bad outcomes. In the present report, a 43-year-old man with diffuse cutaneous SSc received AHSCT. Despite AHSCT, SSc further progressed with progressive symptomatic heart failure with newly developed concomitant mitral and tricuspid valve insufficiency, thus the patient started on RTX 8 months after AHSCT. Shortly after initiation of RTX, clinical symptoms and organ functions ameliorated subsequently. Heart valve regurgitations were reversible after initiation of RTX treatment. Currently, the patient remains in a stable condition with significant improvement of clinical symptoms and organ functions. Reporting about therapies after AHSCT in SSc is a very important issue, as randomized controlled trials are lacking, and therefore this report adds to evidence that RTX can be considered as a treatment option in patients with SSc that do not respond to AHSCT.

scholarly journals Relevance of prognostic factors in the decision-making of stem cell transplantation in Myelodysplastic Syndromes

2016 ◽  
Vol 62 (suppl 1) ◽  
pp. 25-28
Author(s):  
Fernando Barroso Duarte ◽  
Talyta Ellen de Jesus dos Santos ◽  
Maritza Cavalcante Barbosa ◽  
Jacques Kaufman ◽  
João Paulo de Vasconcelos ◽  
...  
Keyword(s):  
Decision Making ◽  
Stem Cell ◽  
Prognostic Factors ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Myelodysplastic Syndrome ◽  
Cell Transplantation ◽  
Myelodysplastic Syndromes ◽  
Limiting Factors ◽  
Hematopoietic Stem

ABSTRACT The hematopoietic stem cell transplantation (HSCT) is the only curative alternative for Myelodysplastic Syndrome (MDS), but many patients are not eligible for this treatment, as there are several limiting factors, especially in the case of patients with low-risk MDS. The aim of this study is to discuss the factors that can guide the decision-making on referring or not a patient to HSCT. Three cases of MDS, two of which were submitted to HSCT are presented. We intend to report the difficulties in referring patients with MDS to transplant and the prognostic factors that contribute to define eligibility.

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