Approximately 20,000 hematopoietic cell transplantation (HCT) procedures are performed in the United States annually. With advances in transplantation technology and supportive care practices, HCT has become safer and patient survival continues to improve over time. Indications for HCT continue to evolve as research refines the role for HCT in established indications and identifies emerging indications where HCT may be beneficial. The American Society for Blood and Marrow Transplantation (ASBMT) established a multi-stakeholder task force consisting of transplant experts, payer representatives and a patient advocate to provide guidance on ‘routine’ indications for HCT. This white paper presents the recommendations from the Task Force. Indications for HCT were categorized as (1) Standard of care, where indication for HCT is well defined and supported by evidence, (2) Standard of care, clinical evidence available, where large clinical trials and observational studies are not available but HCT has been shown to be effective therapy, (3) Standard of care, rare indication, for rare diseases where HCT has demonstrated effectiveness but large clinical trials and observational studies are not feasible, (4) Developmental, for diseases where pre-clinical and/or early phase clinical studies show HCT to be a promising treatment option, and (5) Not generally recommended, where available evidence does not support the routine use of HCT. The ASBMT will periodically review these guidelines and will update them as new evidence becomes available.

Hematopoietic cell transplantation (HCT) is an expensive, resource-intensive, and medically complicated modality for treatment of many hematologic disorders. A well-defined care coordination model through the continuum can help improve health care delivery for this high-cost, high-risk medical technology. In addition to the patients and their families, key stakeholders include not only the transplantation physicians and care teams (including subspecialists), but also hematologists/oncologists in private and academic-affiliated practices. Initial diagnosis and care, education regarding treatment options including HCT, timely referral to the transplantation center, and management of relapse and late medical or psychosocial complications after HCT are areas where the referring hematologists/ oncologists play a significant role. Payers and advocacy and community organizations are additional stakeholders in this complex care continuum. In this article, we describe a care coordination framework for patients treated with HCT within the context of coordination issues in care delivery and stakeholders involved. We outline the challenges in implementing such a model and describe a simplified approach at the level of the individual practice or center. This article also highlights ongoing efforts from physicians, medical directors, payer representatives, and patient advocates to help raise awareness of and develop access to adequate tools and resources for the oncology community to deliver well-coordinated care to patients treated with HCT. Lastly, we set the stage for policy changes around appropriate reimbursement to cover all aspects of care coordination and generate successful buy-in from all stakeholders.

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The use of high dose chemotherapy followed by autologous hematopoietic stem cell transplantation for remission consolidation after initial induction represents standard of care for patients with multiple myeloma. Patients with myeloma and Acquired von Willebrand Syndrome (AVWS) undergoing autologous stem cell transplant (ASCT) are at significant risk of bleeding due to the profound thrombocytopenia, low Factor VIII levels, fever, and toxicities associated with the preparative regimen. We report a patient with AVWS associated with multiple myeloma who underwent autologous stem cell transplants as consolidation after initial induction and again at relapse. He was successfully treated with high dose intravenous immunoglobulin (IVIG) prior to each transplant with rapid resolution of AVWS.

Bundled payments for hematopoietic cell transplantation (HCT) have long been accepted by both commercial health insurance providers and transplant centers, effectively outpacing the use of this payment model elsewhere in health care. As with the rest of health care, interest in payment and health delivery reform has created demand for transplant providers to address value by incorporating quality metrics and strategic changes in network design The complexity of evaluating performance in HCT complicates the goal of rewarding providers for better performance and penalizing poor results. We provide an introduction to value-based purchasing and address potential considerations in the adoption of incentives to improve quality of care in HCT.

between UCB and MMUD for bacterial infections (p ¼ .18) nor fungal infections (p ¼ .52); however, UCB recipients had significantly more viral infections compared to MMUD (p <.0001).The rate (infections per patient per year) of CMV reactivation/disease and adenoviral infections was higher in UCB recipients; MMUD had a higher rate of EBV reactivation (Table 2). Infection as a cause of death was similar among the three graft sources at 1 year (41%, 31%, and 38% for UCB, MUD, and MMUD respectively). Overall survival at 1 year was 50% (46 e 55%), 69% (65 e 72%), and 58% (52 e 64%) for UCB, MUD, and MMUD respectively (p <.0001) and this difference was driven by higher treatment related mortality (TRM) as relapse was similar across the groups. The survival was slightly higher with MMUD compared to UCB (p<.04). In conclusion, 1) The incidences of bacterial, fungal, and particularly viral infections are high after alternative donor transplant; 2) Infections are a major cause of death in the first year after alternative donor transplant; 3) The incidence of infection as cause of death was similar among the graft sources; 4) Viral infections are higher in recipients of UCB compared to MMUD. Future directions will focus on preventative techniques and predictors for infection, particularly for viral infections.

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Fifteen patients with Philadelphia chromosome (Ph)-positive chronic myelogenous leukemia (CML) who were ineligible for allogeneic bone marrow transplantation (BMT) or alpha-interferon therapy were included in this study. Eight patients were in the first late chronic phase, five were in the second chronic phase, one was in the accelerated phase, and one was in the blastic phase. Autologous bone marrow cells (median, 2.5 x 10(8) nucleated cells/kg) were stored at a median of 30 months after diagnosis. Patients were treated with cyclophosphamide (1.5 g/m2 daily for 4 days), carmustine (BCNU) (300 mg/m2), and etoposide (VP-16) (250 mg/m2 daily for 3 days) (CBV), followed by reinfusion of autologous bone marrow. Hematopoietic recovery was rapid, and toxicity was mild to moderate in 14 patients. One patient died of cytomegalovirus pneumonitis. Eight of 15 patients showed Ph suppression to less than 90% Ph-positive metaphases after autologous BMT. Major cytogenetic responses (Ph suppression to less than 35% Ph-positive metaphases) developed in four patients. Cytogenetic responses were observed in 4 of 11 patients infused with 100% Ph-positive marrows, and in all 4 patients infused with Ph-mosaic marrows (mixture of diploid and Ph-positive cells). Better results were observed when autologous BMT was performed in the chronic phase compared with the advanced phases. The major cytogenetic responses have lasted for 3, 4, 12, and 15+ months, whereas minor cytogenetic responses lasted for only a short time (less than 2 months). Three of seven patients (43%) in the chronic phase with previous resistance to alpha-interferon therapy became sensitive to alpha-interferon therapy after autologous BMT. The authors concluded that intensive chemotherapy followed by autologous BMT produced cytogenetic remissions in patients with Ph-positive CML and reinduced disease sensitivity to alpha-interferon therapy in patients previously resistant to it. This is particularly useful when treatment is given during the chronic phase and stem cells are collected at a time of previous cytogenetic remission.