Hematopoietic Stem Cell Transplantation for Treatment of Multiple Sclerosis in Sweden

Summary

This is an observational cohort study with retrospective analysis of prospectively collected data. The study cohort is constituted of all patients with relapsing-remitting multiple sclerosis (RRMS) treated with autologous stem cell transplantation (AHSCT) in Sweden from 2004 when the first AHSCT was performed until 31 December 2019. The study aims to describe the effectiveness, safety and patient reported outcomes of AHSCT for MS through real world data. Treatment related mortality will be analyzed from start of mobilization until the end of the study. For other adverse events the data collection will end 3 months post-transplantation. A statistical subgroup comparison of efficacy and safety between the conditioning regimens BEAM-ATG and Cy-ATG will be included within the study.

Conditions

• Multiple Sclerosis
• Multiple Sclerosis, Relapsing-Remitting

Interventions

PROCEDURE

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.

Sponsors & Collaborators

• Karolinska University Hospital

  collaborator OTHER

• Sahlgrenska University Hospital

  collaborator OTHER

• Uppsala University Hospital

  collaborator OTHER

• Skane University Hospital

  collaborator OTHER

• University Hospital, Linkoeping

  collaborator OTHER

• University Hospital, Umeå

  collaborator OTHER

• Region Örebro County

  collaborator OTHER

• Uppsala University

  lead OTHER

Principal Investigators

• Joachim Burman, MD, PhD · Uppsala University

• Thomas Silfverberg, MD · Uppsala University

Eligibility

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2021-05-05

Primary Completion

2022-09-30

Completion

2022-09-30

Countries

• Sweden

Study Locations