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Hematopoietic stem-cell transplantation, which is used to treat both malignant and nonmalignant conditions, was first conceived more than 50 years ago, but problems associated with transplanting a nonsolid organ and modulating the immune response had to be solved before the procedure could be used clinically. This review summarizes background information about hematopoietic stem-cell transplantation and discusses the current role of the procedure. This review summarizes background information about hematopoietic stem-cell transplantation and discusses the current role of the procedure. Although hematopoietic stem-cell transplantation was originally conceived more than 50 years ago as a treatment for injury from irradiation and, later, for cancer, associated problems needed to be solved before the procedure could be used clinically. Bone marrow, the source of hematopoietic stem cells, is not a solid organ but is rather diffuse and not directly accessible. Furthermore, hematologic cells can initiate immune reactions that may thwart transplantation. Hematopoietic stem-cell transplantation is used primarily for hematologic and lymphoid cancers but also for many other disorders (Table 1). In this review, I summarize background information about hematopoietic stem-cell transplantation and discuss . . .

We investigated the impact of the number of induction/consolidation cycles on outcomes of 3113 adult AML patients who received allogeneic hematopoietic cell transplantation (allo-HCT) between 2008 and 2019. Patients received allo-HCT using myeloablative (MAC) or reduced-intensity (RIC) conditioning in first complete remission (CR) or with primary induction failure (PIF). Patients who received MAC allo-HCT in CR after 1 induction cycle had 1.3-fold better overall survival (OS) than 2 cycles to CR and 1.47-fold better than ≥3 cycles. OS after CR in 2 or ≥3 cycles was similar. Relapse risk was 1.65-fold greater in patients receiving ≥3 cycles to achieve CR. After RIC allo-HCT, the number of induction cycles to CR did not affect OS. Compared to CR in 1 cycle, relapse risk was 1.24-1.41-fold greater in patients receiving 2 or ≥3 cycles. For patients receiving only 1 cycle to CR, consolidation therapy prior to MAC allo-HCT was associated with improved OS vs. no consolidation therapy. Detectable MRD at the time of MAC allo-HCT did not impact outcomes while detectable MRD preceding RIC allo-HCT was associated with an increased risk of relapse. For allo-HCT in PIF, OS was significantly worse than allo-HCT in CR after 1–3 cycles.

Hematopoietic stem-cell transplantation, which is used to treat both malignant and nonmalignant conditions, was first conceived more than 50 years ago, but problems associated with transplanting a nonsolid organ and modulating the immune response had to be solved before the procedure could be used clinically. This review summarizes background information about hematopoietic stem-cell transplantation and discusses the current role of the procedure.

Fatal fulminant hepatic failure (FHF) associated with thalidomide (Thalomid, Celgene Corp, Warren, NJ) therapy has not been reported previously, to our knowledge. We describe a case in which a woman receiving thalidomide therapy for multiple myeloma experienced fatal FHF.

Allogeneic hematopoietic stem cell transplantation provides the most powerful antileukemic effect in the treatment of acute myeloid leukemia. Due to its significant morbidity and mortality, it should be used in first remission patients whose relapse risk is substantial. Reduced intensity transplantation is safer and extends the application of early transplantation to older patients and those with comorbidities. In patients with advanced disease, allotransplantation provides a lower chance for cure, but is often the only curative treatment available. Advances in histocompatibility typing and supportive care have improved results of allogeneic transplantation in acute myeloid leukemia.

The efficacy of reduced intensity conditioning (RIC) allogeneic hematopoietic cell transplantation (HCT) for Philadelphia chromosome positive (Ph+) acute lymphoblastic leukemia (ALL) is uncertain. We analyzed 197 adults with Ph+ ALL in first complete remission; 67 patients receiving RIC were matched with 130 receiving myeloablative conditioning (MAC) for age, donor type and HCT year. Over 75% received pre-HCT tyrosine kinase inhibitors (TKIs), mostly imatinib; 39% (RIC) and 49% (MAC) were minimal residual disease (MRD) neg pre-HCT. At a median 4.5 years follow-up, 1-year transplant-related mortality (TRM) was lower in RIC (13%) than MAC (36%; P =0.001) while the 3-year relapse rate was 49% in RIC and 28% in MAC ( P =0.058). Overall survival (OS) was similar (RIC 39% (95% confidence interval (CI) 27–52) vs 35% (95% CI 27–44); P =0.62). Patients MRD pos pre-HCT had higher risk of relapse with RIC vs MAC (hazard ratio (HR) 1.97; P =0.026). However, patients receiving pre-HCT TKI in combination with MRD negativity pre-RIC HCT had superior OS (55%) compared with a similar MRD population after MAC (33%; P =0.0042). In multivariate analysis, RIC lowered TRM (HR 0.6; P =0.057), but absence of pre-HCT TKI (HR 1.88; P =0.018), RIC (HR 1.891; P =0.054) and pre-HCT MRD pos (HR 1.6; P =0.070) increased relapse risk. RIC is a valid alternative strategy for Ph+ ALL patients ineligible for MAC and MRD neg status is preferred pre-HCT.

Emily E. Johnston, MD, assistant professor and member of the Institute of Cancer Outcomes and Survivorship in the division of pediatric , hematology-oncology at University of Alabama, Birmingham, and colleagues also observed variations in end-of-life care according to patients' age, underlying diagnosis and comorbidities at the time of HSCT. [...]we do not know if this is the end-of-life care these patients and families wanted. [...]it is critical to do follow-up studies to better understand end-of-life goals for patients who die after stem cell transplant, particularly the high-risk groups identified in the study.\" \"Palliative care involvement is associated with lower-intensity end-of-life care in adolescents and young adults with cancer and palliative care integration into inpatient stem cell transplant teams improves symptoms and psycho - logic distress,\" Johnston said. [...]routine use of palliative care in stem cell transplant, particularly high-risk groups, may both decrease medical intensity and improve quality of life of patients undergoing stem cell transplant.\"

An altered version of the Hopkins conditioning regimen may lead to more stable donor engraftment without severe toxicity among patients with sickle cell disease who undergo haploidentical hematopoietic stem cell transplantation, according to study data. \"Less than 20% of sickle cell disease patients will have an HLA-matched donor and alternative stem cell sources, such as haploidentical donors, can expand the donor pool,\" Santosh L. Saraf, MD, assistant professor of medicine at University of Illinois at Chicago, told HemOnc Today. The other eight patients received a modified version of the Hopkins protocol - 30 mg/m2 fludarabine, 14.5 mg/kg cyclophosphamide, 3 Gy total body irradiation, 0.5 mg/kg antithymocyte globulin, 50 mg/kg after transplant cyclophosphamide and infused mobilized peripheral blood stem cells.

A reduced-intensity conditioning regimen yielded comparable RFS and OS as standard myelo ablative conditioning among patients with myelodysplastic syndrome, according to results of the phase 3 RICMAC clinical trial. Reduced-intensity conditioning also improved OS (HR = 0.41; 95% CI, 0.19-0.87). Because the results of the RICMAC trial are based on the standard therapy of busulfan/cyclophosphamide - which is linked to higher transplant-related mortality - more data are needed, Michael A. Pulsipher, MD, section head of blood and marrow transplantation at Children's Center for Cancer and Blood Diseases at Children's Hospital Los Angeles, wrote in a related editorial. Would a young, high-risk patient with MDS with an HLA-identical sibling be entered on this study or simply proceed with a myeloablative transplant? Because the precise criteria for HSCT are not defined, might patients at low risk for relapse undergoing transplantation on this trial diminish the potential relapse-lowering advantage of myeloablation?