José Mauro Kutner

Haploidentical bone marrow transplantation with post transplant cyclophosphamide for patients with X-linked adrenoleukodystrophy: a suitable choice in an urgent situation

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 150u2009mg/m2, cyclophosphamide 29u2009mg/kg and 2u2009Gy total body irradiation (TBI) with or without antithymocyte globulin. Graft-vs.-host disease prophylaxis consisted of cyclophosphamide 50u2009mg/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 2u2009Gy TBI for 4u2009Gy 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.

Platelet aggregation and lipoprotein levels in a patient with familial hypercholescholesterolemia after selective LDL-apheresis

Platelet aggregation was studied in a patient with familial hypercholesterolemia immediately after apheresis selective for low-density lipoprotein (LDL), a lipid-lowering procedure. This treatment reduced plasmatic levels of total and LDL-cholesterol, apo B, and triglyceride. Increased platelet aggregation was reduced immediately after the apheresis in whole blood as well as in platelet-rich plasma. However, aggregation in washed platelets remained unchanged after LDL-apheresis. In conclusion, in this patient reduction of LDL-cholesterol improved platelet function in the very short term.

The impact of preapheresis white blood cell count on autologous peripheral blood stem cell collection efficiency and HSC infusion side effect rate

Autologous peripheral blood hematopoietic stem cell (PBSC) collection efficiency (CE) is reportedly affected by the patients blood properties; however, studies to identify factors correlated with CE have shown inconsistent results. Additionally, variables such as stem cell graft granulocyte content and patient age, sex, and underlying disease, may be associated with hematopietic stem cell (HSC) infusion‐related adverse reactions. In this study, we evaluated the correlation of preleukapheresis PB granulocyte count and PBSC harvest variables with CD34+collection yield and efficiency, and thawed HSC infusion side effect occurrence.

Hematological approaches to multiple myeloma: trends from a Brazilian subset of hematologists. A cross-sectional study

CONTEXT AND OBJECTIVEnFor the last nine years, hematologists and oncologists have gathered annually at an educational symposium organized by a Brazilian and an American hospital. During the 2015 Board Review, a survey among the attendees evaluated the differences in management and treatment methods for multiple myeloma (MM).nnnDESIGN AND SETTINGnCross-sectional study during an educational hematology symposium in São Paulo, Brazil.nnnMETHODSnHematologists present at the symposium gave responses to an electronic survey by means of mobile phone.nnnRESULTSnAmong the 350 attendees, 217 answered the questionnaire. Most of the participants believed that immunotargeting agents (iTA) might be effective for slowing MM progression in heavily pretreated patients (67%) and that continued exposure to therapy might lead to emergence of resistant clones in patients with MM (76%). Most of the physicians use maintenance therapy after hematopoietic stem cell transplantation (95%) and 45% of them would further restrict it to post-transplantation patients with underlying high-risk disease. The first-line drugs used for transplantation-ineligible patients (TI-MM) were bortezomib-thalidomide-dexamethasone (31%), bortezomib-dexamethasone (28%), lenalidomide-dexamethasone (Rd; 17%) and melphalan-based therapy (10%). Lenalidomide was the drug of choice for post-transplantation maintenance for half of the participants. No significant differences were observed regarding age or length of experience.nnnCONCLUSIONnThe treatment choices for TI-MM patients were highly heterogenous and the melphalan-based regimen represented only 10% of the first-line options. Use of maintenance therapy after transplantation was a common choice. Some results from the survey were divergent from the evidence in the literature.