Immunosuppressive Therapy and Future Response to Androgens or Survival After Hematopoietic Stem Cell Transplantation in Fanconi Anemia.
Bibliographic record
Abstract
Abstract Abstract 1082 Poster Board I-104 Introduction A large proportion of patients with Fanconi anemia (FA) develop aplastic anemia (AA), which is unlikely to respond to immune suppressive therapy (IST). It is not known whether exposure to IST might have a negative effect on patients' response to other therapeutic interventions or their cancer risk. We therefore surveyed FA patients in the United States and Canada to determine whether treatment of AA with IST (perhaps inadvertently prior to the diagnosis of FA) jeopardized subsequent response to anabolic steroids or survival following hematopoietic stem cell transplantation (HSCT), or increased the risk of a malignancy. Methods With the help of Fanconi Anemia Research Fund (FARF) and Fanconi Canada (FC), we sent surveys to 905 adults including patients and their parents or other care providers. A single primary respondent was identified for each patient, using the following priority order: mother, father, adult patient or other care provider; each patient was thus accounted for only once. The survey ascertained age, gender, reason for diagnosis of FA, vital status, treatment, HSCT outcome and development of cancer or leukemia. IST was defined as treatment with cyclosporine A, antithymocyte globulin, or intravenous immunoglobulin. The frequencies of androgen responders, HSCT survivors, and malignancy among FA patients who received IST were compared with those who did not receive IST. Results We received responses representing 224 FA patients. The median age (range) of the patients was 17.3 years (2.6-54.4), the male:female ratio was 114:110, and the overall survival was 144/224 (64%). Single, double or pancytopenia led to the diagnosis of FA in 138 of the 224 patients (62%). Fifty-eight of the 224 patients received IST (26%), 74 received androgens (33%), 121 underwent HSCT (54%), and 23 developed a malignancy (10%). Fifteen patients had solid tumors and 9 had leukemia. One patient developed both a solid tumor and leukemia. Twenty-three of 30 (77%) who received androgens after IST reported a response, compared with 51 of 58 (88%) androgen recipients who had not received IST (p = 0.2). Thirty-two of 53 (60%) patients who had prior IST survived HSCT, while 45 of 68 (66%) without IST for AA also survived HSCT (p = 0.6). Three of 58 (5%) patients developed a malignancy after IST compared with 20 of 166 (12%) without IST (p = 0.2). Conclusion Treatment of AA in a patient who is subsequently diagnosed with FA does not appear to have negative or positive effects on later response to androgens, survival following HSCT, or the risk of malignancy. The limitations of this study are its retrospective nature, the inclusion of multiple family members as the respondents, the inability to ascertain the timing of IST, and its dependence on self-report. The apparent lack of not only harmful but beneficial effects of IST raises questions about its utility for the treatment of AA in FA patients, and suggests that the use of IST may lead to delay in appropriate management. 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 | 0.002 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.000 |
| Bibliometrics | 0.000 | 0.001 |
| 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".