Real-world outcomes in adult histiocytosis: the uncharted territory of orphan diseases
Bibliographic record
Abstract
Adult histiocytic disorders are rare, and limited data exists on spectrum, clinical presentation and real-world management strategies. Here, we present Canadian single-center experience spanning over 23 years. Ninety-six patients were identified, with Langerhans Cell Histiocytosis (LCH; n = 60), Erdheim-Chester disease (ECD; n = 11); Rosai-Dorfman disease (RDD; n = 19) constituting the majority. At a median follow-up of 38 months, median OS of our cohort was not reached with encouraging 5-year OS of 96.3%, 79.5% and 86% amongst LCH, ECD and RDD patients respectively. Although, 5-year PFS rates were higher for LCH and RDD compared to ECD patients (61.7% vs 47% vs 24.2%; p = 0.320), these were not statistically significant. LCH patients with RO involvement had inferior 2-year PFS compared to those without (33.3% vs 75.8%; p = 0.042). A high incidence of concomitant hematological malignancies, (n = 3/11 in ECD and n = 3/60 in LCH) in our cohort warrants the need for further translational studies to explore possible association.
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 | 0.003 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.000 |
| Bibliometrics | 0.000 | 0.001 |
| Science and technology studies | 0.001 | 0.001 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.000 | 0.001 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.001 | 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".