Hematopoietic Stem Cell Transplantation for Systemic Sclerosis: If You Are Confused, Remember: “It Is a Matter of the Heart”
Bibliographic record
Abstract
For the past 20 years, the standard of care for systemic sclerosis (SSc) with lung involvement has been oral or intravenous (IV) cyclophosphamide (CYC). To date, there have been 2 randomized trials and 2 metaanalyses of prospective studies using oral or IV CYC in SSc-related interstitial pneumonitis (interstitial lung disease; ILD) and none have reported improvement in lung function1,2,3,4,5. The Scleroderma Lung Research Study Group’s study in The New England Journal of Medicine reported that oral CYC daily for 1 year is of “modest benefit” compared to placebo1. However, the term “modest benefit” does not mean that lung function improved. In fact, the forced vital capacity (FVC) and DLCO declined in both placebo and CYC-treated patients1. “Modest benefit” means that, after 1 year, the rate of decline in FVC was less in those receiving CYC compared to placebo, but the lung function still worsened on CYC1. Further, at 2-year followup there was no difference in loss of lung function between oral daily CYC and placebo2. Due to a lack of effective standard therapy, SSc — a lethal disease that involves vital organs — needs a new and effective approach. An approach that began in patients about 14 years ago, hematopoietic stem cell transplantation (HSCT), has been demonstrated to improve both skin and lung function as well as quality of life in patients with SSc6,7,8. Transplantation has been performed safely in some studies8, but results have been complicated by high treatment-related mortality in others9. Mortality of HSCT for SSc can be markedly reduced, however, if the reasons for mortality are properly recognized. The safety of HSCT is determined by 3 variables: (1) … Address correspondence to Dr. Burt; E-mail: rburt{at}northwestern.edu
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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 | 0.003 | 0.009 |
| Meta-epidemiology (narrow) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.001 | 0.001 |
| Science and technology studies | 0.001 | 0.001 |
| Scholarly communication | 0.002 | 0.005 |
| Open science | 0.001 | 0.001 |
| Research integrity | 0.005 | 0.009 |
| Insufficient payload (model declined to judge) | 0.014 | 0.010 |
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".