Studies have shown that autologous hematopoietic SCT (HSCT) can be used as an intensive immunosuppressive therapy to treat refractory patients and to prevent the progression of multiple sclerosis (MS). This is a prospective multicentric Brazilian MS trial comparing two conditioning regimens: BEAM/horse ATG and CY/ rabbit ATG. Most (80.4%) of the 41 subjects in the study had the secondary progressive MS subtype and the mean age was 42 years. The baseline EDSS score in 58.5% of the subjects was 6.5 and 78% had a score of 6.0 or higher, respectively. The complication rate during the intratransplantation period was 56% for all patients: 71.4% of the patients in the BEAM/hATG group and 40% in the CY/rATG group (P ¼ 0.04). Three subjects (7.5%) died of cardiac toxicity, sepsis and alveolar hemorrhage, all of them in the BEAM/ATG group. EFS was 58.54% for a ll patients: 47% in the BEAM/hATG group and 70% in the CY/rATG group (P ¼ 0.288). In conclusion, the CY/rATG regimen seems to be associated with similar outcome results, but presented less toxicity when compared with the BEAM/hATG regimen. Long-term followup would be required to fully assess the differences in therapeutic effectiveness between the two regimens.

Moderate to severe chronic graft-versus-host disease (GVHD) is treated with potent immunosuppressive therapy (IST) to modulate the allo-immune response, control symptoms, and prevent further organ damage. We sought to understand the types of treatments used in clinical practice and the likelihood of successful treatment associated with each. A chart review was performed for 250 adult patients at Fred Hutchinson Cancer Research Center enrolled in a prospective observational study. After a median follow-up of 5.6 years for survivors, approximately one-third were still on IST (of whom half were on fourth or greater line of therapy), one-third were alive and off IST, and one-third had relapsed or died. Approximately half of survivors stopped all IST at least once, although half of these restarted IST after a median of 3.4 months (interquartile range, 2.3 to 8.0) off therapy. Successful discontinuation of IST for at least 9 months was associated with myeloablative conditioning (P = .04), more years since transplant (P = .009), and lack of oral (P < .001) and skin (P = .049) involvement compared with those who had to restart IST. We conclude that patients with chronic GVHD usually receive multiple lines and years of IST, with only a third off IST, alive, and free of malignancy at 5 years after chronic GVHD diagnosis. Patients stopping IST should be cautioned to self-monitor and continue close medical follow-up, especially for 3 to 6 months after stopping IST.

The formula of Pierelli et al. is associated with a PPV that is high, moderate, and relatively low for the corresponding predicted CD34+ doses.

Allogeneic hematopoietic stem cell transplantation (HSCT) is the only treatment that enhances survival and stabilizes neurologic symptoms in X-linked adrenoleukodystrophy (X-ALD) with cerebral involvement, a severe demyelinating disease of childhood. Patients with X-ALD who lack a well-matched HLA donor need a rapid alternative. Haploidentical HSCT using post transplant cyclophosphamide (PT/Cy) has been performed in patients with malignant and nonmalignant diseases showing similar outcomes compared to other alternative sources. We describe the outcomes of transplants performed for nine X-ALD patients using haploidentical donors and PT/Cy. Patients received conditioning regimen with fludarabine 150 mg/m, cyclophosphamide 29 mg/kg and 2 Gy total body irradiation (TBI) with or without antithymocyte globulin. Graft-vs.-host disease prophylaxis consisted of cyclophosphamide 50 mg/kg/day on days +3 and +4, tacrolimus or cyclosporine A and mycophenolate mofetil. One patient had a primary graft failure and was not eligible for a second transplant. Three patients had secondary graft failure and were successfully rescued with second haploidentical transplants. Trying to improve engraftment, conditioning regimen was changed, substituting 2 Gy TBI for 4 Gy total lymphoid irradiation. Eight patients are alive and engrafted (17-37 months after transplant). Haploidentical HSCT with PT/Cy is a feasible alternative for X-ALD patients lacking a suitable matched donor. Graft failure has to be addressed in further studies.

Objectives: To evaluate the results of the comprehensive geriatric assessment (CGA) before allogeneic hematopoietic stem cell (HSCT) transplantation in patients aged 60 years and over. Methods: We evaluated all consecutive patients undergoing CGA before HSCT between September 2011 and July 2018 in a private hospital in Brazil. We also evaluated the Hematopoietic Cell Transplantation-Specific Comorbidity Index (HCT-SCI) and the Disease Risk Index (DRI). Results: During the study period, 61 patients were referred for transplant evaluation. After exclusions, we analyzed 40 patients, with a mean age of 67.6 years (60-76). The CGA detected vulnerability and frailty in 43% and 18.9% respectively according to the Fried Frailty Phenotype score; limitations across the domain of function and disability with handgrip test alterations in 65.8%. However, 36 (90%) were independent for instrumental activities of daily living (IADL). Cognitive and depression domain have shown abnormal with the clock test in 44.4%, and loss of memory complains in 37.5%. But the mini-mental test was normal in 89%. Geriatric Depression Scale (GDS) was normal in 82.5%. 30% were considered at risk for malnutrition. Half of the patients (50%) had a high Hematopoietic Cell Transplantation Comorbidity Index (HCT-CI) score. 32.5% needed ICU admission. The overall survival and non-relapse mortality at 2 years were 41.8% and 38.7% respectively. Conclusion: The CGA was feasible in detecting the patients' vulnerabilities in our population. More studies, multicentric and with a larger number of patients, are needed to evaluate the role of CGA in this context of allo-HCT in our population.

No abstract

No abstract

No abstract