Paroxysmal Nocturnal Hemoglobinuria and Marrow Failure Treated by Infusion of Marrow from an Identical Twin
Bibliographic record
Abstract
An 18-year-old white Canadian male patient with paroxysmal nocturnal hemoglobinuria and refractory marrow failure received a marrow infusion from his normal identical twin brother without prior ablation of the patient's marrow by drugs or irradiation. After 2 years of follow-up, the patient is well with no evidence of clinical disease or significant hematological abnormality. The results suggest that normal marrow stem cells can have a selective advantage over the abnormal paroxysmal nocturnal hemoglobinuria clone. PAROXYSMAL NOCTURNAL HEMOGLOBINURIA is a rare dis-order of the erythrocyte membrane that may be charac-terized by chronic hemolysis, intermittent hemoglobinuria and hemosiderinuria, venous thrombic phenomena, and marrow hypoplasia. The disease reflects the establishment of a clone of abnormal marrow stem cells. The clinical and laboratory features have been reviewed elsewhere (1-4). We have followed a patient with paroxysmal nocturnal hemoglobinuria and refractory marrow failure who was unique in having a normal genetically identical twin (5). Because eight examples have been reported (6, 7) in which aplastic anemia was successfully treated simply by infusion of marrow from a normal twin, this approach was adopted for the patient with paroxysmal nocturnal hemoglobinuria. We hoped that infused normal marrow stem cells would repopulate the patient's hemopoietic system and function normally despite the absence of any therapy directed against the abnormal paroxysmal noctur-nal hemoglobinuria clone. The initial presentation and clinical course of the patient before marrow infusion has been reported (5). The patient was admitted for marrow transplantation in June 1973 because of a 9-month history of progressive marrow failure, pancytopenia, recurrent hemolytic crises, and life-threatening infections despite conventional therapy. Subsequent follow-up investigations, last done in July 1975, documented a progressive and continued clinical and hema-tologic remission.
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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 | 0.001 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.001 | 0.001 |
| 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".