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Cytokine-based mobilization in light chain (AL) amyloidosis is frequently complicated by fluid overload, weight gain, cardiac arrhythmias, and peri-mobilization mortality. We analyzed hematopoietic progenitor cells (HPC) mobilization outcomes in 49 consecutive AL amyloidosis patients at our institution between 2004 and 2013 with granulocyte colony-stimulating factor (G) (10 μg/kg/day) (n = 25) versus an institutional protocol to limit G exposure using plerixafor (P) (.24 mg/kg s.c. starting day 3 of G 10 μg/kg) (n = 24). G+P strategy yielded higher total CD34(+) cells/kg (12.8 × 10(6) versus 6.3 × 10(6); P.001) and CD34(+) cells/kg collected on day 1 (10.8 × 10(6) versus 4.9 × 10(6), P = .004) compared with the G cohort. More G+P patients collected ≥5 × 10(6) CD34(+) HPCs/kg (22 versus 16, P = .02) and ≥ 10 × 10(6) CD34(+) HPCs/kg (13 versus 5, P = .01). Four patients (16%) had mobilization failure with G; none with G+P. Peri-mobilization weight gain was lower with G+P strategy (median weight gain 1 versus 7 pounds, P = .009). Numbers of apheresis sessions (median, 1 versus 1, P = .52), number of hospitalization days (median, 1.1 versus 1.6, P = .52), transfusions, use of intravenous antibiotics, and cardiac arrhythmias were similar. In conclusion, our study demonstrates that upfront use of G+P as a mobilization strategy results in superior HPC collection, no mobilization failures, and less weight gain than G alone.

Hematopoietic cell transplantation (HCT) is an intensive treatment for hematologic malignancies that has the potential to cure disease or prolong life, but also to impair quality of life for survivors. Earlier studies have suggested that various factors are associated with physical and mental health after HCT. In this study, we evaluated demographic and clinical factors before and after HCT and selected psychosocial factors after HCT, exploring their association with self-reported physical and mental health. We studied a cohort of 662 survivors at a median of 6.6 years after HCT. Pre-HCT demographic and clinical factors accounted for only a small amount of the variance in physical and mental health post-HCT (3% and 1%, respectively). Adding post-HCT clinical variables to the pre-HCT factors accounted for 32% and 7% of physical and mental outcomes, respectively. When both clinical and psychosocial factors were considered, better physical health post-HCT was associated with younger age, race other than white, higher current family income, currently working or being a student, less severe transplantation experience (ie, not experiencing graft-versus-host disease), fewer current comorbidities, higher Karnofsky status, less social constraint, less social support, and less trait anxiety. This multivariate model accounted for 36% of the variance in physical health, with the psychosocial variables contributing very little. When both clinical and psychosocial factors were considered, better mental health after HCT was associated with more severe transplantation experience, less social constraint, greater spiritual well being, and less trait anxiety. This multivariate model accounted for 56% of the variance in mental health, with the psychosocial factors accounting for most of the variance. These data suggest that clinical factors are explanatory for much of the post-HCT physical health reported by HCT survivors, but very little of self-perceived mental health. These observations provide insight into the identification of factors that can allow recognition of at-risk patients, as well as factors amenable to intervention.

Blacks are twice as likely to develop and die from multiple myeloma (MM) and are less likely to receive an autologous hematopoietic-cell transplant (AHCT) for MM compared to whites. The influence of race on outcomes of AHCT for MM is not well described. We compared the probability of overall survival, progression-free survival, disease progression and non-relapse mortality among black (N=303) and white (N=1892) recipients of AHCT for MM, who were reported to the Center for International Blood and Marrow Transplant Research (CIBMTR) from 1995 to 2005. The black cohort was more likely to be female, had better Karnofsky performance scores but lower hemoglobin and albumin levels at diagnosis. Black recipients were younger and more likely to be transplanted later in their disease course. Disease stage and treatment characteristics prior to AHCT were similar between the two groups. Black and white recipients had similar probabilities of 5-year overall survival (52% vs. 47%, P=0.19) and progression-free survival (19% vs. 21%, P=0.64) as well as cumulative incidences of disease progression (72% vs. 72%, P=0.97) and non-relapse mortality (9% vs. 8%, P=0.52). In multivariate analyses, race was not associated with any of these endpoints. Black recipients of AHCT for MM have similar outcomes compared to whites, suggesting that the reasons underlying lower rates of AHCT in blacks need to be studied further to ensure equal access to effective therapy.

Clinical research examining the role of hematopoietic stem cell transplantation (SCT) in the therapy of acute myeloid leukemia (AML) in children is presented and critically evaluated in this systematic evidence-based review. Specific criteria were used for searching the published literature and for grading the quality and strength of the evidence and the strength of the treatment recommendations. Treatment recommendations based on the evidence are presented in the table entitled "Summary of Treatment Recommendations Made by the Expert Panel for Pediatric Acute Myeloid Leukemia" and were reached unanimously by a panel of experts in AML. The identified priority areas of needed future research in pediatric AML include: What is the role of risk group stratification, including the role of cytogenetics, in selection of patients for allogeneic SCT, especially those in first CR? What is the appropriate timing and use of alternative donor SCT, given that matched unrelated donor SCT appears to yield outcomes equivalent to matched related donor SCT? What is the role of reduced intensity SCT (including the use of fludarabine-based preparative regimens) and/or other immunomodulatory approaches to maximize the graft-versus-leukemic effect? and What is the role of biologically targeted agents (ie, tyrosine kinase inhibitors, farnesyl transferase inhibitors, Flt-3 inhibitors, etc) in the treatment of AML, including induction, consolidation, conditioning regimens, and after SCT?

Hematopoietic cell transplantation (HCT) is a highly specialized and resource-intense medical procedure that can be associated with disparities in access to transplantation. Barriers to access to HCT are multifactorial, complex, and interrelated. Our current knowledge of specific barriers that prevent access to HCT is very limited. As the utilization of HCT increases, it is imperative that underserved populations receive the benefit of this life-saving procedure. We review the prevailing literature on access to HCT and describe research priorities for eliminating disparities in transplantation. Better understanding of these complex barriers will minimize inequities, inform health policy, guide development of interventions targeted to eliminate disparities, and continue the expansion of HCT in the future.

We have reported a lower incidence of acute graft-versus-host disease (aGVHD) with a novel conditioning regimen using low-dose rabbit antithymocyte globulin (ATG; Thymoglobulin [TG]) with fludarabine and intravenous busulfan (FluBuTG). To assess further this single-center experience, we performed a retrospective matched-pair analysis comparing outcomes of adult patients transplanted using the FluBuTG conditioning regimen with matched controls from patients reported to the CIBMTR receiving a first allogeneic hematopoietic stem cell transplant (HCT) after standard oral busulfan and cyclophosphamide (BuCy). One hundred twenty cases and 215 matched controls were available for comparison. Patients receiving FluBuTG had significantly less treatment related mortality (TRM; 12% versus 34%, P < .001) and grades II-IV aGVHD (15% versus 34%, P < .001) compared to BuCy patients. The risk of relapse was higher in the FluBuTG patients (42% versus 20%, P < .001). The risks of chronic GVHD (cGVHD) and disease free survival (DFS) were similar in the cases and controls. These results suggest that the novel regimen FluBuTG decreases the risk of aGVHD and TRM after HLA-identical sibling HSCT, but is associated with an increased risk of relapse, resulting in similar DFS. Whether these conditioning regimens may be more suitable for specific patient populations based on relapse risk requires testing in prospective randomized trials.

Shortage of manpower and center capacity is expected to be a major challenge to the anticipated future growth in the utilization of allogeneic hematopoietic cell transplantation (HCT) in the United States. Using data from the National Marrow Donor Program's Transplant Center Network Renewal Survey, we describe transplant center and transplant physician capacity in the United States from 2005 to 2009. Over this 5-year period, the number of allogeneic transplants increased by 30%, bed capacity increased by 17%, and physician full-time equivalents increased by 26%. The number of related donor HCT increased by 15% and unrelated donor HCT increased by 45%. In addition to large centers, small- and medium-sized centers also made a major contribution to overall national transplant volumes for both related and unrelated donor HCT. Increase in utilization of unrelated donor HCT occurred in centers irrespective of their size. The majority of transplant centers were performing more transplantations using existing physician and bed capacity. Our study provides important descriptions of allogeneic transplant activity and capacity of U.S. centers, and our data will assist policy makers plan for the projected growth in the use of transplantation.

Despite prophylaxis with immunosuppressive agents or a variety of other approaches, many patients suffer from acute graft-versus-host disease after allogeneic hematopoietic cell transplantation. Although consensus has emerged supporting the use of high-dose methylprednisolone or prednisone for initial treatment of acute GVHD, practices differ among centers with respect to the initial glucocorticoid dose, the use of additional immunosuppressive agents, and the approach to withdrawal of treatment after initial improvement. Despite many studies, practices vary considerably with respect to the selection of agents for treatment of glucocorticoid-resistant or refractory GVHD. Investigators and clinicians have recognized the lack of progress and lamented the absence of an accepted standard of care for secondary treatment of acute GVHD. The American Society of Blood and Marrow Transplantation has developed recommendations for treatment of acute GVHD to be considered by care providers, based on a comprehensive and critical review of published reports. Since the literature provides little basis for a definitive guideline, this review also provides a framework for the interpretation of previous studies and the design of future studies.

Studies show that engaging patients in exercise and/or stress management techniques during hematopoietic cell transplantation (HCT) improves quality of life. The Blood and Marrow Transplant Clinical Trials Network tested the efficacy of training patients to engage in self-directed exercise and stress management during HCT. The study randomized 711 patients at 21 centers to receive 1 of 4 training interventions before HCT: a self-directed exercise program, a self-administered stress management program, both, or neither. Participants completed self-reported assessments at enrollment and up to 180 days after HCT. Randomization was stratified by center and transplant type. There were no differences in the primary endpoints of the Physical Component Summary and Mental Component Summary scales of the Medical Outcomes Study Short Form 36 at day +100 among the groups, based on an intention-to-treat analysis. There also were no differences in overall survival, days of hospitalization through day +100 post-HCT, or in other patient-reported outcomes, including treatment-related distress, sleep quality, pain, and nausea. Patients randomized to training in stress management reported more use of those techniques, but patients randomized to training in exercise did not report more physical activity. Although other studies have reported efficacy of more intensive interventions, brief training in an easy-to-disseminate format for either self-directed exercise or stress management was not effective in our trial.

Success of hematopoietic cell transplantation (HCT) can vary by race, but the impact of socioeconomic status (SES) is not known. To evaluate the role of race and SES, we studied 6207 unrelated-donor myeloablative (MA) HCT recipients transplanted between 1995 and 2004 for acute or chronic leukemia or myelodysplastic syndrome (MDS). Patients were reported by transplant center to be White (n = 5253), African American (n = 368), Asian/Pacific-Islander (n = 141), or Hispanic (n = 445). Patient income was estimated from residential zip code at time of HCT. Cox regression analysis adjusting for other significant factors showed that African American (but not Asian or Hispanic) recipients had worse overall survival (OS) (relative-risk [RR] 1.47; 95% confidence interval [CI] 1.29-1.68, P < .001) compared to Whites. Treatment-related mortality (TRM) was higher in African Americans (RR 1.56; 95% CI 1.34-1.83, P < .001) and in Hispanics (RR 1.30; 95% CI 1.11-1.51, P = .001). Across all racial groups, patients with median incomes in the lowest quartile (

In 1999, the American Society for Blood and Marrow Transplantation (ASBMT) began the development of systematic evidence-based reviews and position statements on the effectiveness of autologous and allogeneic hematopoietic stem cell transplantation (SCT) for specific diseases. The purpose of these reviews is to achieve broader and more consistent coverage from payers for established indications for SCT, and provide evidence in support of clinical decisions and matters of public policy regarding SCT. The ASBMT Evidence-Based Review Steering Committee, chaired by Dr. Roy Jones, developed a policy outlining the methodology to be followed for these reviews [1]. Evaluation of this process after the completion of the first two reviews resulted in a revised policy statement in 2005 [2], which focused the review process to address questions most relevant to clinicians and scientists. Currently, seven reviews have been published in Biology of Blood and Marrow Transplantation on the use of SCT in the therapy of: diffuse large B-cell non-Hodgkin lymphoma (NHL) [3], multiple myeloma (MM) [4], pediatric acute lymphoblastic leukemia (ALL) [5], adult ALL [6], pediatric acute myelogenous leukemia (AML) [7], adult AML [8], and myelodysplastic syndromes (MDS) [9].