Autologous Hematopoietic Stem Cell Transplantation in Neuromyelitis Optica - An Update (P5.259)
Bibliographic record
Abstract
Objective: To determine if Neuromyelitis Optica patients, despite maintenance therapy, experience a reduction in relapses and disability without ongoing immunosuppressants after autologous non-ablative hematopoietic stem cell transplantation (AHSCT). We hypothesize => 50[percnt] reduction in the proportion of patients relapsing 3 years post-transplant. Background: Neuromyelitis Optica (NMO) is an immune-mediated astrocytopathy characterized by frequent demyelinating/necrotizing attacks of optic nerves and spinal cord. Treatments are highly toxic and incompletely 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. Patients undergo non-ablative stem cell mobilization and infusion using cyclophosphamide, rituximab and ATG. Outcomes include EDSS, NMO-IgG titers, OCT and MRI q6-12 months for 5 years. Ten subjects provide 80[percnt] power for our primary outcome. Results: Three NMO patients have undergone transplantation thus far. 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 at month 41 respectively. A 36F was transplanted in April 2012 with pre-transplant ARR of 5, now 1 and 3.5 at month 30 respectively, who started on mycophenolate mofetil at her request. A 39M was transplanted in January 2014 with pre-transplant ARR of 1.3 and EDSS 3.5 who remains stable at 10 months. Despite clinical improvement in all patients, the latter two patients, both NMO-IgG seropositive, remain seropositive. 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.
Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.
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 | 0.001 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.000 |
| Bibliometrics | 0.001 | 0.001 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.001 | 0.001 |
| Insufficient payload (model declined to judge) | 0.003 | 0.001 |
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".