Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
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Biol. Blood Marrow Transplant. · Jul 2017
Ruxolitinib as Salvage Therapy in Steroid-Refractory Acute Graft-versus-Host Disease in Pediatric Hematopoietic Stem Cell Transplant Patients.
We describe our retrospective clinical experience with ruxolitinib for steroid-refractory acute graft-versus-host disease (GVHD) in pediatric allogeneic hematopoietic stem cell transplant (HSCT) patients. Ruxolitinib was administered orally at 5 mg twice daily for children ≥ 25 kg or 2.5 mg twice daily if <25 kg. We excluded patients who received new immune suppressive agents within 2 weeks before initiation of ruxolitinib from response analysis. ⋯ Seven of 13 patients were alive at a median follow-up of 401 days (range, 219 to 969) after HSCT. We observed a high rate of reversible adverse effects in children with steroid-refractory acute GVHD and a fair overall response of ruxolitinib as a salvage therapeutic agent. Further pharmacokinetic studies are needed to determine the best-tolerated dose of ruxolitinib that will achieve efficacy without significant adverse effects.
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Biol. Blood Marrow Transplant. · Jun 2017
A Phase I Trial of High-Dose Lenalidomide and Melphalan as Conditioning for Autologous Stem Cell Transplantation in Relapsed or Refractory Multiple Myeloma.
Autologous stem cell transplantation (ASCT) conditioned with high-dose chemotherapy has long been established as the standard of care for eligible patients with newly diagnosed multiple myeloma. Despite recent therapeutic advances, high-dose melphalan (HDM) remains the chemotherapy regimen of choice in this setting. Lenalidomide (LEN) in combination with low-dose dexamethasone is recognized as a standard of care for patients with relapsed or refractory multiple myeloma (RRMM), and there is growing support for the administration of LEN as maintenance therapy post-ASCT. ⋯ Median progression-free and overall survival were 10 months and 22 months, respectively, in this population of heavily pretreated patients. Our findings suggest that HDLEN in combination with HDM may offer significant potential as a conditioning regimen before ASCT in patients with RRMM. These preliminary findings are now being evaluated further in an ongoing phase II clinical trial.
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Biol. Blood Marrow Transplant. · Jun 2017
Prognostic Index for Critically Ill Allogeneic Transplantation Patients.
Outcomes of intensive care for allogeneic hematopoietic stem cell transplantation (Allo-SCT) patients remain poor. Better selection of critically ill Allo-SCT patients for intensive care may alleviate costs to the patients, families, and the health care system. We aimed to develop a prognostic index tailored for critically ill Allo-SCT patients as traditional instruments are of limited value in this setting. ⋯ Hospital mortality and median overall survival of patients with PICAT scores of 0 to 2 (n = 141), >2 to 4 (n = 242), and >4 (n = 182) were 34%, 69%, and 91%; and 7.59, .67, and .30 months, respectively. PICAT has good calibration and accuracy in predicting mortality for Allo-SCT patients requiring intensive care. Its AUC was significantly higher than APACHE II and SOFA scores and is also associated with overall survival.
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Biol. Blood Marrow Transplant. · Jun 2017
Citrulline and Monocyte-Derived Macrophage Reactivity before Conditioning Predict Acute Graft-versus-Host Disease.
During conditioning, intestinal damage induces microbial translocation which primes macrophage reactivity and leads to donor-derived T cell stimulation. Little is known about the role of intestinal health and macrophage reactivity before conditioning in the development of acute graft-versus-host disease (aGVHD) in patients undergoing allogeneic hematopoietic cell transplantation (allo-HCT). We assessed (1) citrulline, a surrogate marker of functional enterocyte mass and (2) circulating monocyte-derived macrophage reactivity, before allo-HCT. ⋯ In multivariate analysis, IL-10 ≥ 90 pg/mL, myeloablative conditioning, and citrulline ≤20 µmol/L remained independent factors of aGVHD development (hazard ratio [HR], 8.18, P = .0003; HR, 4.28, P = .006; and HR, 4.43, P = .01, respectively). Preconditioning citrulline and monocyte-derived macrophage reactivity are objective surrogate markers suitable to identify patients at risk of developing aGVHD. This work highlights the influence of preconditioning status in aGVHD development.
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Biol. Blood Marrow Transplant. · May 2017
BMT Roadmap: A User-Centered Design Health Information Technology Tool to Promote Patient-Centered Care in Pediatric Hematopoietic Cell Transplantation.
Health information technology (HIT) has great potential for increasing patient engagement. Pediatric hematopoietic cell transplantation (HCT) is a setting ripe for using HIT but in which little research exists. "BMT Roadmap" is a web-based application that integrates patient-specific information and includes several domains: laboratory results, medications, clinical trial details, photos of the healthcare team, trajectory of transplant process, and discharge checklist. BMT Roadmap was provided to 10 caregivers of patients undergoing first-time HCT. ⋯ Caregivers found the tool useful and easy to use, leading them to want even greater access to information. BMT Roadmap was feasible, with no disruption to inpatient care. Although this initial study is limited by the small sample size and single-institution experience, these initial findings are encouraging and support further investigation.