Bone Marrow Transplantation From HLA-Identical Sibling for Thalassemia.
Bibliographic record
Abstract
Abstract Abstract 3361 Poster Board III-249 Allogeneic bone marrow transplantation (BMT) is curative treatment for β-Thalassemia major. The most extensive patient series have been reported by transplant teams in Italy. Outside these large groups, smaller, more limited series have reported variable results. Here we report the current experience of BMT for thalassemia outside Italy, and describe the characteristics and transplant outcomes in children after HLA-identical sibling BMT for thalassemia. Patients aged ≤20 years at BMT who received a myeloablative HLA-matched sibling BMT for thalassemia between 1995 and 2001 at 19 centers were eligible for the survey (n=179). Recipients of mobilized peripheral blood or umbilical cord blood stem cells, and reduced intensity transplant preparation were excluded as there were too few patients in each category for analysis. Also excluded were patients previously reported by the Italian centers. The median age at BMT was 7 years and the median follow-up of survivors was 6 years. Most recipients had high-risk features at BMT. The distribution of Lucarelli risk class I, II and III categories was 2%, 42%; and 36%, respectively. Data required to assign a risk category was not available for 20% of the study population. Before BMT, 85% had received ≥ 20 red blood cell transfusions. Of 87% who had a pre-BMT liver biopsy, 59% had portal fibrosis. There was hepatomegaly in 52% of patients and 94% had inadequate iron chelation therapy. All patients received busulfan and cyclophosphamide with or without anti-thymocyte globulin as the transplant conditioning regimen and almost all received cyclosporine-containing GVHD prophylaxis. The overall survival (OS) probabilities at 100 days and at 5 years were 85% and 80%, respectively. Seventeen patients (9.5%) had graft failure that was fatal in 11 cases. Six of 9 patients with graft failure survive after a successful second transplantation. Thirty-five patients (20%) died after BMT and the leading cause of death was interstitial pneumonitis in the current analysis. The probability of acute GVHD (grade II-IV) was 38% and the 5-year probability of chronic GVHD was 13%. Veno-occlusive disease (VOD) occurred frequently after BMT (n=58; 32% of patients) and 21 of the 35 (57%) patients who died after BMT had had a history VOD. A multivariate analysis showed that age at BMT ≥10 years (RR 2.16, 95% CI 1.1 – 4.23, p=0.025) and baseline hepatomegaly (RR 5.57, 95% CI 2.14 – 14.49, p<0.001) were associated with higher risk of mortality. The probabilities of disease-free survival (DFS) and OS are shown in the Table below. Younger patients without hepatomegaly fared the best and, older patients with hepatomegaly, the worst. This series confirms reports from large single center series that the results after HLA-matched sibling BMT for thalassemia major are optimized when performed in young children before the development of risk-factors for poor outcome. Pulmonary and hepatic complications were the leading cause of death in high-risk patients. 5-year OS 5-year DFS Age < 10 years without hepatomegaly 96% 93% Age < 10 years with hepatomegaly 73% 69% Age ≥ 10 years without hepatomegaly 86% 81% Age ≥ 10 years with hepatomegaly 53% 53% Disclosures: No relevant conflicts of interest to declare.
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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 | 0.000 | 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) | 0.003 | 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".