Bone marrow transplantation from matched unrelated donors for patients with severe combined immune deficiency
Bibliographic record
Abstract
Introduction: Bone Marrow Transplant (BMT) from related HLA identical donor (RID) is the treatment of choice for patients with severe combined immuno-deficiency (SCID). In the absence of RID, bone marrow from HLA haplo-identical (half) related donors (HID) have often been used. However, HID is frequently associated with reduced survival and failure of long-term immune reconstitution. HLA matched unrelated donors (MUD) represent another promising alternative for patients with SCID. Methods: We studied hematopoietic engraftment, occurrence of graft versus host disease, infections and other complication, and survival in infants diagnosed with SCID who received MUD BMT between 1990 and 2004 in a Canadian pediatric referral center specializing in such procedures. Detailed evaluations of immune reconstitution were performed in children that survived more than 2 years after transplant. Results: During the 14 years of this study, 22 infants underwent MUD BMT in our center. Molecular diagnosis was available in 64% of them. All infants received myelo-ablative conditioning pre-transplant. Despite prophylaxis with cyclosporine and methylprednisolone, acute graft versus host disease occurred in 15 of the 22 patients, and it was the most common cause of death. All patients developed complete donor lymphocyte engraftment. None of the 15 patients who are two or more years after transplant requires intravenous immunoglobulin replacement therapy. After discontinuing immune suppression all patients have normal T cell function and T cell repertoire, which is sustained for more than 14 years of follow up. Current survival is 73% and it is even better for patients with mutations in the IL-2 receptor pathway. Furthermore, survival of patients who presented with low B cells, previously estimated to have unfavorable outcome, is not significantly lower than in other forms of SCID in this study. Conclusions: MUD BMT leads to long term survival, engraftment, and immune function in SCID patients and should be the preferred treatment for clinically stable infants who do not have a related HLA-identical donor.
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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 | 0.001 |
| 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".