Publications by authors named "K M Mahadeo"
Article Synopsis
- The study examines the outcomes of hematopoietic cell transplantation (HCT) in infants with Hurler syndrome (HS) diagnosed through newborn screening (NBS).
- Patients underwent a busulfan-based treatment regimen and umbilical cord blood HCT, showing promising results with no graft failures and recovery of enzyme levels.
- At follow-up, most patients were thriving and meeting developmental milestones, suggesting that NBS and early transplantation are effective in treating HS.
Article Synopsis
- Ceroid lipofuscinosis neuronal (CLN) is a rare neurodegenerative disorder that affects children, causing issues like epilepsy, vision loss, and early death.
- A study analyzed 8 patients with different types of CLN who underwent umbilical cord blood transplant (UCBT) between 2012 and 2020, finding that all patients had successful donor cell engraftment without severe transplant-related deaths.
- Complications like severe graft-versus-host disease and infections were noted, but overall, UCBT showed promise in stabilizing some patients' conditions, highlighting the need for more research and longer follow-up to fully understand its impact.
Patients undergoing immune effector cell therapy (IECT) are at high risk for infections. We assessed seropositivity against pneumococcus, tetanus, and diphtheria in patients before and after IECT and the patients' response to vaccination. We enrolled patients who underwent IECT from January 2020 to March 2022.
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- Aberrant expression of HOX and MEIS1 family genes in certain leukemias disrupts normal blood cell differentiation and contributes to leukemia development.
- Menin inhibitors can target the interaction between KMT2A and menin, reducing the abnormal expression of key factors and promoting differentiation in these leukemias.
- A collaborative effort among pediatric and adult specialists aims to advance menin inhibitors in treatment, offering a comprehensive overview of clinical trials and advocating for inclusive trial designs for youth.