autologous transplant to end nmo spectrum disorder

Conditions: Neuromyelitis Optica; Devic's Disease; NMO Spectrum Disorder

Summary: this study is designed to treat your disease with an autologous stem cell transplant using a regimen of immune suppressant drugs and chemotherapy to reset your immune system and to determine if your disease will go into long-term remission.

the autologous stem cell transplant used in this research study is an investigational procedure that uses cyclophosphamide (chemotherapy), rabbit antithymocyte globulin (ratg) (a protein that kills the immune cells that are thought to be causing your disease), rituximab (a biologic drug that targets b cells of your immune system), and intravenous immunoglobulin (ivig) (pooled igg antibodies from plasma donors with immunomodulatory and anti-inflammatory effects), followed by return of your own previously collected blood stem cells (autologous stem cell transplant). one day of plasmapheresis will also be performed the day prior to admission for stem cell transplant to remove disease-causing antibodies. the ability of this experimental treatment to stop relapses and progression (worsening) of your nmosd will be assessed.

Eligibility criteria:
inclusion criteria: 1. age 18 - 65 years old at the time of pre-transplant evaluation 2. an established diagnosis of nmosd (with or without aquaporin 4 (aqp4)-igg antibody) exclusion criteria: 1. under age of 18 or over age of 65 2. prisoners 3. psychiatric illness or mental deficiency making compliance with treatment or informed consent impossible, or any adult who is unable to consent (for adults cognitively impaired due to disease, consent may be obtained from the closest living relative). 4. paraplegia or quadriplegia (must be able to use a walker if even for only a few feet) 5. extensive subcortical white matter lesions 6. uncontrolled diabetes mellitus or any other illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive treatment 7. myocardial infarction within the last 12 months. if longer than 12 months, must pass a dobutamine stress test and be cleared by cardiology. 8. active systemic lupus erythematous, sjogren's, myasthenia gravis, or another autoimmune disease 9. sickle cell disease, sickle cell disease, or coagulopathy 10. prior history of malignancy that required any radiotherapy, chemotherapy, or biological therapy 11. positive pregnancy test, inability or unable to pursue effective means of birth control, or failure to willingly accept or comprehend irreversible sterility as a side effect of therapy 12. women who are breastfeeding 13. untreated life-threatening cardiac arrhythmia on electrocardiogram (ekg) or 24-hour holter 14. left ventricular ejection fraction (lvef) \<50% 15. tiffeneau-pinelli index (fev1/fvc) \<70% of predicted after bronchodilator therapy (if necessary), or diffusing capacity of lung for carbon monoxide (dlco) hemoglobin corrected \<70 % predicted 16. serum creatinine \>2.0 mg/dl 17. liver cirrhosis, transaminases \>2x of normal limits, or bilirubin \>2.0 mg/dl unless due to gilbert's disease 18. major hematological abnormalities such as platelet count \< 100,000/μl or absolute neutrophil count (anc) \< 1000/μl 19. active infection except asymptomatic bacteriuria 20. presence of metallic objects implanted in the body that would preclude the ability of the patient to safely have magnetic resonance imaging (mri) exams 21. known hypersensitivity to mouse, rabbit, or e. coli derived proteins 22. human immunodeficiency virus (hiv) positive 23. hepatitis b or c positive 24. use of natalizumab (tysabri) within the previous six months 25. use of fingolimod (gilenya) within the previous three months 26. use of dimethyl fumarate (tecfidera) within the previous three months 27. use of teriflunomide (aubagio) unless cleared from the body (plasma concentration \<0.02mcg/ml) following elimination from the body with cholestyramine 8g three times a day for 11 days 28. use of alemtuzumab (lemtrada/campath) within previous 12 months 29. use of rituximab (rituxan) or ocrelizumab (ocrevus) within previous six months 30. prior treatment with mitoxantrone (novantrone)

Keywords: autologous stem cell transplantation hematopoietic stem cell transplant