Autologous Hematopoietic Stem Cell Transplantation in Neuromyelitis Optica (P4.001)
Bibliographic record
Abstract
OBJECTIVE: To determine if Neuromyelitis Optica patients, despite maintenance therapy, experience a reduction in relapse and disability without need for immunosuppressants after non-ablative autologous hematopoietic stem cell transplantation (AHSCT). We hypothesize => 50% reduction in the proportion of patients relapsing 3 years post-transplant. BACKGROUND: Neuromyelitis Optica (NMO) is an immune-mediated neurodegenerative disease of the CNS characterized by frequent demyelinating/necrotizing attacks of optic nerves and spinal cord. Treatments are highly toxic and only minimally effective. Fifty percent of patients are blind in one eye or require a mobility aide within 5 years. The immunological features of NMO, coupled with its severity make it an ideal candidate for a trial of AHSCT with the goal of disease remission and freedom from immunosuppressive medications. DESIGN/METHODS: Patients 18-65 with => 1 relapse in 12 months or => 2 in 24 months despite immunotherapy and EDSS < 6.5 are eligible for enrolment. Patients undergo non-ablative stem cell mobilization and infusion using cyclophosphamide, rituximab and ATG. Outcomes include EDSS, NMO-Ig titers, OCT and MRI q6-12 months for 5 years. Ten subjects will provide 80% power for our primary outcome. RESULTS: Two NMO patients have undergone transplantation. A 28F was transplanted in May 2011 with pre-transplant annualized relapse rate (ARR) of 5 and EDSS 4.5, now 0 and 2.0 respectively. A 36F was transplanted in April 2012 with pre-transplant ARR of 5, now 0 and 3.5 respectively, with one presumed pseudorelapse without EDSS change treated with high-dose steroids. No additional rescue or maintenance therapy has been required. CONCLUSIONS: Thus far, this AHSCT regimen is well-tolerated with marked clinical improvement. Additional patients and longer follow-up will determine if these benefits persist. Study Supported by:
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.001 | 0.000 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.000 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.002 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".