Endocrine Sequelae in Pediatric Hematopoietic Stem Cell Transplant Recipients with Sickle Cell Disease: A Retrospective Cohort Analysis
Bibliographic record
Abstract
Background: The NIH non-myeloablative regimen has been successfully implemented for pediatric sickle cell disease (SCD) patients undergoing matched sibling donor (MSD) hematopoietic stem cell transplant (HSCT) in an effort to prevent late complications, including infertility and other endocrine sequelae. This retrospective cohort analysis evaluated the prevalence of endocrine complications in 17 pediatric SCD patients who underwent non-myeloablative MSD HSCT. Procedure: Medical records between June 2013 and June 2020 were reviewed. Data was extracted from baseline and 1,2,3, and 5-years post HSCT. Results: There were 12 females and 5 males. Follicle stimulating hormone (FSH) elevation post HSCT occurred in 42.8%; 4 females and 2 males. All females with elevated FSH had subsequent normalization of their values with time. FSH elevation in males did not resolve. Post HSCT secondary amenorrhea or oligomenorrhea was described in 4 females; however, improvement or resolution occurred in all. One female subject with normal gonadotrophin levels post HSCT had a successful pregnancy and live birth. Vitamin D deficiency (100% when measured), and obese or overweight body mass index post HSCT (41.2%) were also reported. Conclusions: A notable endocrine issue post HSCT described in this cohort is FSH elevation. The elevation was transient in females, and we identified one successful pregnancy, suggesting that non-myeloablative conditioning may convey favorable fertility outcomes compared to busulfan-based conditioning. Not all patients had baseline endocrine evaluations or consistent post HSCT endocrine testing. We recommend standardizing pre- and post HSCT endocrinology assessments for this population.
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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 | 0.001 | 0.001 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.000 |
| Bibliometrics | 0.001 | 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) | 0.001 | 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".