scholarly journals Advising patients seeking stem cell interventions for multiple sclerosis

2018 ◽  
Vol 18 (6) ◽  
pp. 472-476 ◽  
Author(s):  
Beatrice von Wunster ◽  
Steven Bailey ◽  
Alastair Wilkins ◽  
David I Marks ◽  
Neil J Scolding ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Treatment Options ◽  
Progressive Multiple Sclerosis ◽  
Current Treatment ◽  
Haematopoietic Stem Cell ◽  
Financial Risks ◽  
Safety Standards

Given the intuitive potential of stem cell therapy and limitations of current treatment options for progressive multiple sclerosis (MS), it is not surprising that patients consider undertaking significant clinical and financial risks to access stem cell transplantation. However, while increasing evidence supports autologous haematopoietic stem cell transplantation (AHSCT) in aggressive relapsing–remitting MS, interventions employing haematopoietic or other stem cells should otherwise be considered experimental and recommended only in the context of a properly regulated clinical study. Understandably, most neurologists are unfamiliar with AHSCT procedures and the specific requirements for quality assurance and safety standards, as well as post-procedure precautions and follow-up. Consequently they may feel ill-equipped to advise patients. Here, we highlight important points for discussion in consultations with patients considering stem cell ‘tourism’ for MS.

scholarly journals Autologous haematopoietic stem cell transplantation fails to stop demyelination and neurodegeneration in multiple sclerosis

Brain ◽  
2007 ◽  
Vol 130 (5) ◽  
pp. 1254-1262 ◽  
Author(s):  
I. Metz ◽  
C. F. Lucchinetti ◽  
H. Openshaw ◽  
A. Garcia-Merino ◽  
H. Lassmann ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Haematopoietic Stem Cell Transplantation ◽  
Haematopoietic Stem Cell

scholarly journals Autologous haematopoietic stem cell transplantation for treatment of multiple sclerosis

2017 ◽  
Vol 13 (7) ◽  
pp. 391-405 ◽  
Author(s):  
Paolo A. Muraro ◽  
Roland Martin ◽  
Giovanni Luigi Mancardi ◽  
Richard Nicholas ◽  
Maria Pia Sormani ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Haematopoietic Stem Cell Transplantation ◽  
Haematopoietic Stem Cell

scholarly journals Rescue treatment with eltrombopag in refractory cytopenias after allogeneic stem cell transplantation

2020 ◽  
Vol 11 ◽  
pp. 204062072096191
Author(s):  
Semra Aydin ◽  
Chiara Dellacasa ◽  
Sara Manetta ◽  
Luisa Giaccone ◽  
Laura Godio ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Treatment Options ◽  
Graft Function ◽  
Complete Response ◽  
Haematopoietic Stem Cell ◽  
Poor Graft Function ◽  
Engraftment Failure ◽  
Grade 3

Background: Patients with post-transplant cytopenias due to poor graft function or primary engraftment failure show poor prognosis with a high mortality rate mainly because of graft versus host disease (GVHD), infection and/or bleeding. Treatment options are scarce and a CD34+ stem cell boost or a second bone marrow transplantation may be required to restore adequate haematopoiesis. Methods: In the present study patients with primary engraftment failure ( n = 1) and refractory poor graft function ( n = 11) were treated with eltrombopag in a single centre. The reason for eltrombopag treatment was trilineage cytopenia in six patients, bilineage cytopenia in three patients and single lineage cytopenia in three patients. Eltrombopag was initiated at a median of 214 (range: 120–877) days after haematopoietic stem cell transplantation (HCST) and administered for a median time of 114 (range: 12 days to >490) days. In 8/12 patients eltrombopag was introduced at a dose of 75 mg/day and then increased to 150 mg/day after 1 week; 1 patient was given 50 mg eltrombopag per day, and 3 patients received 75 mg daily. Results: In 10/12 patients eltrombopag significantly enhanced blood count values and patients became transfusion independent. Once stable haematological response was obtained, treatment was tapered until final discontinuation in 9/10 responding patients. No grade 3 or 4 toxicities were observed. At time of last follow up, 3/12 patients were dead, 2 due to disease relapse, 1 due to GVHD and pneumonia. All patients except one maintained their complete response and remain transfusion independent at a median of 858 (range: 429–1119) days. Conclusion: These preliminary data confirm that eltrombopag is able to rescue multilineage haematopoiesis in patients with treatment-refractory cytopenias after allogeneic HSCT.

scholarly journals A prospective, randomized, controlled trial of autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: a position paper

2012 ◽  
Vol 18 (6) ◽  
pp. 825-834 ◽  
Author(s):  
R Saccardi ◽  
MS Freedman ◽  
MP Sormani ◽  
H Atkins ◽  
D Farge ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Haematopoietic Stem Cell Transplantation ◽  
Controlled Trial ◽  
Case Series ◽  
Haematopoietic Stem Cell ◽  
Controlled Clinical Trial ◽  
Randomized Controlled

Background: Haematopoietic stem cell transplantation (HSCT) has been tried in the last 15 years as a therapeutic option in patients with poor-prognosis autoimmune disease who do not respond to conventional treatments. Worldwide, more than 600 patients with multiple sclerosis (MS) have been treated with HSCT, most of them having been recruited in small, single-centre, phase 1–2 uncontrolled trials. Clinical and magnetic resonance imaging outcomes from case series reports or Registry-based analyses suggest that a major response is achieved in most patients; quality and duration of response are better in patients transplanted during the relapsing–remitting phase than in those in the secondary progressive stage. Objectives: An interdisciplinary group of neurologists and haematologists has been formed, following two international meetings supported by the European and American Blood and Marrow Transplantation Societies, for the purpose of discussing a controlled clinical trial, to be designed within the new scenarios of evolving MS treatments. Conclusions: Objectives of the trial, patient selection, transplant technology and outcome assessment were extensively discussed. The outcome of this process is summarized in the present paper, with the goal of establishing the background and advancing the development of a prospective, randomized, controlled multicentre trial to assess the clinical efficacy of HSCT for the treatment of highly active MS.

scholarly journals A Global Study of Haematopoietic Stem Cell Transplantation in Multiple Sclerosis and Other Neurodegenerative Disorders

2021 ◽  
Author(s):  
Peter Panegyres ◽  
Jodi Russell ◽  
Huei-Yang Chen
Keyword(s):  
Multiple Sclerosis ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Treatment Protocol ◽  
The United States ◽  
Internet Survey ◽  
Haematopoietic Stem Cell ◽  
Haemopoietic Stem Cell ◽  
Haemopoietic Stem Cell Transplantation

Abstract Aim: The aim of this research is to understand the uptake of haemopoietic stem cell transplantation (HSCT) in neuroimmunological disorders like multiple sclerosis (MS). Methods: A global internet survey was conducted of people having had HSCT, comparing demographics, treatment protocol, and effectiveness. Results: Of 271 participants useful data was available in 223. 73.5% were female in the age range of 35-54 years, most had a household income greater than US$50,000 and the majority of participants were from Australia and the United States of America. 94.6% had MS. Most had their treatment in Russia (38.7%) and 78.1% had non-myeloablative transplants. Nearly half of the participants spent between US$50,000 to US$74,999. 54.5% of neurologists were not supportive of their patients having HSCT. 85.5% of participants believed HSCT helped them manage their disease from weeks to years after transplantation. 9.45% would recommend the treatment. The average reduction in Expanded Disability Status Score after transplantation was 1.2 [95% CI; 0.97-1.41; N=197; p < 0.0001; t: 10.7, df 196]. Conclusions: Participants were supportive of HSCT despite the costs and would recommend it to others. The data suggest some benefit in minimizing disability in MS and provides justification for large randomized controlled trials.

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