Hematopoietic Stem Cell Transplantation for Treatment of Multiple Sclerosis in Sweden
NCT ID: NCT05029206
Last Updated: 2023-11-22
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
174 participants
OBSERVATIONAL
2021-05-05
2022-09-30
Brief Summary
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Detailed Description
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Baseline data will be collected from the SMSreg. Data concerning AHSCT will be collected from local repositories of the EBMT and supplemented by data obtained by reviewing of medical records. This includes data such as doses and names of drugs used for mobilization and conditioning, dates for administration of these drugs, date of hematopoietic stem cell transplantation, date of hematological milestones, occurrence and grading of adverse events during the first three months after the intervention.
Data on clinical outcome after the first three months of the intervention will be collected from SMSreg. Data on vital status will be collected from medical records at the end of study. Any recorded deaths will be analyzed through the medical records to determine if it was treatment-related.
The endpoints will be analysed and described for the whole study cohort. A subgroup analysis comparing the outcome of patients treated with different conditioning regimens (e.g. BEAM-ATG and Cy-ATG) will be included in this study.
Conditions
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Study Design
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COHORT
RETROSPECTIVE
Interventions
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Autologous hematopoietic stem cell transplantation
The therapeutic intervention of AHSCT consists of four parts: the mobilization of hematopoietic stem cells (HSC), the harvest of HSC, the ablation (conditioning) of the immune system and the reinfusion of autologous HSCs.
1. In Sweden, the mobilization of HSCs has been made by a combination of cyclophosphamide (2 g/m2) and granulocyte-colony-stimulating factor.
2. A minimum of 2 × 10\^6 CD34+ cells/kg is harvested and cryopreserved. No in vitro manipulation is done to the stem cells.
3. Two conditioning regimens have been used in Sweden for ablation. The BEAM-ATG protocol include carmustine (BCNU) 300 mg/m2, etoposide 800 mg/m2, cytarabine arabinoside (ARA-C) 800 mg/m2 and melphalan 140 mg/m2 + rATG or hATG. The Cy-ATG protocol include cyclophosphamide 200 mg/kg + rATG/hATG with 1000 mg Metylprednisolone given day -5 to -1.
4. After a minimum of 24 hours after the last administration of chemotherapy have passed, the reinfusion of autologous CD34+ cells is performed.
Eligibility Criteria
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Inclusion Criteria
* Autologous hematopoietic stem cell transplantation performed for treating multiple sclerosis at a Swedish transplantation center until December 31st 2019.
Exclusion Criteria
* Patient not accepted reporting of data to the EBMT register.
* Not fulfilling requirements of the minimal dataset, se below.
Definition of minimal dataset
* Data on disease course of multiple sclerosis at the time of transplantation.
* Transplantation and the following in-patient care performed in Sweden.
* Date of transplantation.
* Data on drugs used in conditioning.
* At least one follow-up visit performed in Sweden (unless early death before first follow-up visit) including data on:
* Clinical assessment
* The Kurtzke Expanded Disability Status Scores (EDSS)
Additional note: For a patient to be included in the analysis of treatment effectiveness data on MRI evaluation is needed at least once during follow-up.
ALL
No
Sponsors
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Karolinska University Hospital
OTHER
Sahlgrenska University Hospital
OTHER
Uppsala University Hospital
OTHER
Skane University Hospital
OTHER
University Hospital, Linkoeping
OTHER
University Hospital, Umeå
OTHER
Region Örebro County
OTHER
Uppsala University
OTHER
Responsible Party
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Principal Investigators
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Joachim Burman, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Uppsala University
Thomas Silfverberg, MD
Role: STUDY_CHAIR
Uppsala University
Locations
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Sahlgrenska University Hospital
Gothenburg, , Sweden
Linköping University Hospital
Linköping, , Sweden
Skåne University Hospital
Lund, , Sweden
Örebro University Hospital
Örebro, , Sweden
Danderyd Hospital
Stockholm, , Sweden
Karolinska University Hospital
Stockholm, , Sweden
Umeå University Hospital
Umeå, , Sweden
Uppsala University Hospital
Uppsala, , Sweden
Countries
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References
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Other Identifiers
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EPM 2021-01530
Identifier Type: -
Identifier Source: org_study_id