Standard of Care Guidelines and Resource Links

ASBMT Education Committee Standard of Care Guidelines and Resource Links

Purpose

This resource is an online collection of citations for key publications in the field of hematopoietic stem cell transplantation. The purpose is to provide practitioners with key literature sources that support the practice of hematopoietic stem cell transplantation in adult patients.

Each citation is listed in standard American Medical Association format and contains the web link to the PubMed abstract as well as the PubMed identification number for ease of access. The citations contained have been selected and reviewed by members of the ASBMT Pharmacy Special Interest Group (SIG) Education Committee and have been updated through October 2015.

 

ADULT

AUTOLOGOUS STEM CELL TRANSPLANTATION REFERENCES

OVERVIEW OF AUTOLOGOUS STEM CELL TRANSPLANTATION

OVERVIEW OF AUTOLOGOUS STEM CELL TRANSPLANTATION

DISEASE SPECIFIC OUTCOMES AUTOLOGOUS STEM CELL TRANPSLANTATION

Diffuse Large B-Cell Lymphoma

Follicular Lymphoma


Mantle Cell Lymphoma

 

T-Cell Lymphoma

 

Hodgkin Lymphoma

 

Multiple Myeloma

 

ALLOGENEIC STEM CELL TRANSPLANT REFERENCES

OVERVIEW OF ALLOGENEIC STEM CELL TRANSPLANTATION

 

MYELOABLATIVE CONDITIONING

  • Multi-Institutional Study of Post-Transplantation Cyclophosphamide As Single-Agent Graft-Versus-Host Disease Prophylaxis After Allogeneic Bone Marrow Transplantation Using Myeloablative Busulfan and Fludarabine Conditioning
  • Conditioning therapy with intravenous busulfan and cyclophosphamide (IV BuCy2) for hematologic malignancies prior to allogeneic stem cell transplantation: a phase II study
  • Once-daily intravenous busulfan given with fludarabine as conditioning for allogeneic  stem cell transplantation: study of pharmacokinetics and early clinical outcomes
  • Conditioning with fludarabine and targeted busulfan for transplantation of allogeneic hematopoietic stem cells
  • Cyclophosphamide combined with antithymocyte globulin in preparation for allogeneic marrow transplants in patients with aplastic anemia
  • Long-term follow-up of a randomized trial comparing the combination of cyclophosphamide with total body irradiation or busulfan as conditioning regimen for patients receiving HLA-identical marrow grafts for acute myeloblastic leukemia in first complete remission.
  • Intravenous busulfan and melphalan, tacrolimus, and short-course methotrexate followed by unmodified HLA-matched related or unrelated hematopoietic stem cell transplantation for the treatment of advanced hematologic malignancies.

T-CELL DEPLETION

  • T-cell-depleted allogeneic bone marrow transplantation as post remission therapy for acute myelogenous leukemia: freedom from relapse in the absence of graft-versus-host disease.
  • Effect of graft-versus-host disease prophylaxis on 3-year disease-free survival in recipients of unrelated donor bone marrow (T-cell Depletion Trial): a multi-centre, randomized phase II-III trial.
  • The history and future of T-cell depletion as graft-versus host disease prophylaxis for allogeneic for allogeneic hematopoietic stem cell transplantation
  • T cell-depleted stem cell transplantation for adults with high-risk acute lymphoblastic leukemia: long-term survival for patients in first complete remission with a decreased risk of graft-versus-host disease
  • T cell-depleted unrelated donor stem cell transplantation provides favorable disease-free survival for adults with hematologic malignancies
  • T cell depleted stem-cell transplantation for adults with hematologic malignancies: sustained engraftment of HLA-matched related donor grafts without the use of antithymocyte globulin
  • Ex vivo T cell-depleted versus unmodified allografts in patients with acute myeloid leukemia in first complete remission
  • Transplantation in remission improves the disease-free survival of patients with advanced myelodysplastic syndromes treated with myeloablative T cell-depleted stem cell transplants from HLA-identical siblings
  • A novel GVHD-prophylaxis with low-dose alemtuzumab in allogeneic sibling or unrelated donor hematopoietic cell transplantation: the feasibility of de-escalation
  • Fludarabine, melphalan, and alemtuzumab conditioning in adults with standard-risk advanced acute myeloid leukemia and myelodysplastic syndrome
  • Fludarabine-melphalan conditioning for AML and MDS: alemtuzumab reduces acute and chronic GVHD without affecting long-term outcomes.

 

NON-MYELOABLATIVE TRANSPLANTATION

  • Transplant-lite: induction of graft-versus-malignancy using fludarabine-based nonablative chemotherapy and allogeneic blood progenitor-cell transplantation as treatment for lymphoid malignancies
  • Nonablative allogeneic hematopoietic transplantation as adoptive immunotherapy for indolent lymphoma: low incidence of toxicity, acute graft-versus-host disease, and treatment-related mortality
  • HLA-matched unrelated donor hematopoietic cell transplantation after nonmyeloablative conditioning for patients with hematologic malignancies.
  • In vivo CAMPATH-1H prevents graft-versus-host disease following nonmyeloablative stem cell transplantation.

REDUCED INTENSITY CONDITIONING

  • Melphalan and purine analog-containing preparative regimens: reduced-intensity conditioning forpatients with hematologic malignancies undergoing allogeneic progenitor cell transplantation.
  • Conditioning therapy with intravenous busulfan and cyclophosphamide (IV BuCy2) for hematologic malignancies prior to allogeneic stem cell transplantation: a phase II study.
  • Nonmyeloablative stem cell transplantation and cell therapy as an alternative to conventional bone marrow transplantation with lethal cytoreduction for the treatment of malignant and nonmalignant hematologic diseases.
  • Reduced intensity conditioning followed by allografting of hematopoietic cells and produce clinical and molecular remissions in patients with poor risk hematologic malignancies.

CORD BLOOD TRANSPLANTATION

  • Umbilical-cord blood transplantation for the treatment of cancer
    • Barker JN, Wagner JE.  Nat Rev Cancer.  2003;3(7):526-32
    • PMID:  12835672
  • Transplantation of 2 partially HLA-matched umbilical cord blood units to enhance engraftment in adults with hematologic malignancy
  • Outcomes after transplantation of cord blood or bone marrow from unrelated donors in adults with leukemia
  • Transplants of Umbilical-Cord Blood or Bone Marrow from Unrelated Donors in Adults with Acute Leukemia.
  • Busulfan/melphalan/antithymocyte globulin followed by unrelated donor cord blood transplantation for treatment of infant leukemia and leukemia in young children: the Cord Blood Transplantation study (COBLT) experience.
  • Outcomes of transplantation of unrelated donor umbilical cord blood and bone marrow in children with acute leukemia: a comparison study
  • Unrelated umbilical cord blood transplant for adult acute lymphoblastic leukemia in first and second complete remission: a comparison with allografts from adult unrelated donors
  • Alternative donor hematopoietic stem cell transplantation for mature lymphoid malignancies after reduced-intensity conditioning regimen: similar outcomes with umbilical cord blood and unrelated donor peripheral blood
  • Double unit grafts successfully extend the application of umbilical cord blood transplantation in adults with acute leukemia
  • Alternative donor transplantation after reduced intensity conditioning: results of parallel phase 2 trials using partially HLA-mismatched related bone marrow or unrelated double umbilical cord blood grafts.

DISEASE-SPECIFIC OUTCOMES FOR ALLOGENEIC STEM CELL TRANSPLANT

Aplastic Anemia

  • Outcome of allogeneic hematopoietic cell transplantation from HLA-identical siblings for severe aplastic anemia in patients over 40 years of age.
  • Retrospective study of alemtuzumab vs ATG-based conditioning without irradiation for unrelated and matched sibling donor transplants in acquired severe aplastic anemia: a study from the British Society for Blood and Marrow Transplantation.
  • Tacrolimus versus cyclosporine after hematopoietic cell transplantation for aquiree aplastic anemia

Myelodysplastic Syndromes (MDS)

  • Unrelated donor marrow transplantation for myelodysplastic syndromes: outcome analysis in 510 transplants facilitated by the National Marrow Donor Program.
  • A decision analysis of allogeneic bone marrow transplantation for the myelodysplastic syndromes: delayed transplantation for low-risk myelodysplasia is associated with improved outcome.
  • Role of reduced-intensity conditioning allogeneic hematopoietic stem cell transplantation in older patients with de novo myelodysplastic syndromes: an international collaborative decision analysis.
  • Impact of donor source on hematopoietic cell transplantation outcomes for patients with myelodysplastic syndromes (MDS)
  • Allogeneic haematopoietic stem cell transplantation in myelodysplastic syndromes.

ACUTE MYLEOID LEUKEMIA (AML)

  • T-cell-depleted allogeneic bone marrow transplantation as postremission therapy for acute myeloid leukemia: freedom from relapse in the absence of graft-versus-host disease.
  • Intravenous busulfan and melphalan, tacrolimus, and short-course methotrexate followed by unmodified HLA-matched related or unrelated hematopoietic stem cell transplantation for the treatment of advanced hematologic malignancies.
  • The role of cytotoxic therapy with hematopoietic stem cell transplantation in the therapy of acute myelogenous leukemia in adults: an evidence-based review.
  • Similar outcomes using myeloablative vs reduced-intensity allogeneic preparative regimens for AML or MDS.
  • Better leukemia-free and overall survival in AML in first remission following cyclophosphamide in combination with busulfan compared with TBI

ACUTE LYMPHOBLASTIC LEUKEMIA (ALL)

  • Allogeneic bone marrow transplantation versus chemotherapy for the treatment of childhood acute lymphoblastic leukemia in second remission: a single-institution study.
  • In adults with standard-risk acute lymphoblastic leukemia, the greatest benefit is achieved from a matched sibling allogeneic transplantation in first complete remission, and an autologous transplantation is less effective than conventional consolidation/maintenance chemotherapy in all patients: final results of the International ALL Trial (MRC UKALL XII/ECOG E2993).
  • Management of adult patients with acute lymphoblastic leukemia in first complete remission: a systematic review and meta-analysis.

 

CHRONIC MYELOID LEUKEMIA (CML)

  • Allogeneic bone marrow transplantation for chronic myeloid leukemia: comparative analysis of unrelated versus matched sibling donor transplantation.
  • European LeukemiaNet recommendations for the management of chronic myeloid leukemia: 2013
  • Allogeneic hematopoietic stem cell transplantation is an effective salvage therapy for patients with chronic myeloid leukemia presenting with advanced disease or failing treatment with tyrosine kinase inhibitors
    • Nair AP, Barnett MJ, Broady RC, et al.  Biol Blood Marrow Transplant 2015 Apr 10.  pii: S1083-8791(15)00249-9. doi: 10.1016/j.bbmt.2015.04.005. [Epub ahead of print]
    • http://www.ncbi.nlm.nih/gov/pubmed/25865648
    • PMID: 25865648

NON-HODGKIN LYMPHOMA (NHL)

  • Nonmyeloablative allogeneic hematopoietic transplantation: a promising salvage therapy for patients with non-Hodgkin’s lymphoma whose disease has failed a prior autologous transplantation.
  • Allotransplantation for patients ≥ 40 years with non-Hodgkin lymphoma: encouraging progression-free survival.
  • A comparison of HLA-identical sibling allogeneic versus autologous transplantation for diffuse large B cell lymphoma: a report from the CIBMTR.
  • Conditioning regimens for allotransplants for diffuse large B-cell lymphoma: myeloablative or reduced intensity?
  • Comparison of reduced-intensity and conventional myeloablative regimens for allogeneic transplantation in non-Hodgkin lymphoma.

HODGKIN LYMPHOMA

  • Reduced-intensity allogeneic stem cell transplantation in relapsed and refractory Hodgkin’s disease: low transplant-related mortality and impact of intensity of conditioning regimen.
  • Role of allogeneic stem cell transplantation in relapsed or refractory Hodgkin’s disease.
  • Allogeneic transplantation improves the overall and progression free survival of Hodgkin lymphoma patients relapsing after autologous transplantation: a retrospective study based on the time of HLA typing and donor availability.

GRAFT-VERSUS- HOST DISEASE PROPHYLAXIS

  • Sirolimus, tacrolimus, and low-dose methotrexate for graft-versus-host disease prophylaxis in mismatched related donor or unrelated donor transplantation.
  • Selective depletion of alloreactive donor lymphocytes: a novel method to reduce the severity of graft-versus-host disease in older patients undergoing matched sibling donor stem cell transplantation.
  • Phase III study comparing methotrexate and tacrolimus (prograf, FK506) with methotrexate and cyclosporine for graft-versus-host disease prophylaxis after HLA-identical sibling bone marrow transplantation.
  • A randomized phase II trial comparing tacrolimus and mycophenolate mofetil to tacrolimus and methotrexate for acute graft-versus-host disease prophylaxis.
  • Tacrolimus/sirolimus vs. tacrolimus/methotrexate as GVHD prophylaxis after matched, related donor allogeneic HCT.
  • The addition of sirolimus to tacrolimus/methotrexate GVHD prophylaxis in children with ALL: a phase 3 Children's Oncology Group/Pediatric Blood and Marrow Transplant Consortium trial.
  • Post-transplant high-dose cyclophosphamide for the prevention of graft-versus-host disease: a review
  • HLA-haploidentical peripheral blood stem cell transplantation with post-transplant cyclophosphamide after busulfan-containing reduced-intensity conditioning.
  • Haploidentical transplant with posttransplant cyclophosphamide vs matched unrelated donor transplant for acute myeloid leukemia.

ACUTE GRAFT-VERSUS-HOST DISEASE

  • In situ dissection of the graft-versus-host activities of cytotoxic T cells specific for minor histocompatibility antigens.
  • An acute graft-versus-host disease activity to predict survival after hematopoietic cell transplantation with myeloablative conditioning regimens.
  • Etanercept, mycophenolate, denileukin, or pentostatin plus corticosteroids for acute graft-versus-host disease: a randomized phase 2 trial from the Blood and Marrow Transplant Clinical Trials Network.
  • Phase III clinical trial steroids/mycophenolate mofetil vs. steroids/placebo as therapy for acute graft-versus-host disease: BMT CTN 0802.
  • Extracorporeal photopheresis in steroid-refractory acute or chronic graft-versus-host disease: Results of a systematic review of prospective studies
  • Extracorporeal photopheresis for treatment of adults and children with acute GVHD: UK consensus statement and review of published literature.
  • Risk factors and prognosis of hepatic acute GVHD after allogeneic hematopoietic cell transplantation.


CHRONIC GRAFT-VERSUS-HOST DISEASE

  • Therapy for chronic-graft-versus-host disease: a randomized trial comparing cyclosporine plus prednisone versus prednisone alone.
  • Failure-free survival after second-line systemic treatment of chronic graft-versus-host disease.
  • Failure-free survival after initial systemic treatment of chronic graft-versus-host disease.
  • National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging workup group report.
  • National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. The 2014 diagnosis and staging working group report.
  • NIH consensus development project on criteria for clinical trials in chronic graft-versus-host disease: II. The 2014 pathology working group report.
  • National Institutes of Health Consensus development project on criteria for clinical trials in chronic graft-versus-host disease: III. The 2014 biomarker working group report.
  • Measuring therapeutic response in chronic graft-versus-host disease. National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: IV. The 2014 response criteria working group report.
  • National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: V. The 2014 ancillary therapy and supportive care working group report.
  • National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: VI. The 2014 clinical trial design working group report.

GRAFT-VERSUS-TUMOR

  • Molecules and mechanisms of the graft-versus-leukemia effect.
  • Generation of minor histocompatibility antigen HA-1 specific cytotoxic T cells restricted by nonself HLA molecules: a potential strategy to treat relapsed leukemia after HLA-mismatched stem cell transplantation.
  • Female donors contribute to a selective graft-versus-leukemia effect in male recipients of HLA-matched, related hematopoietic stem cell transplants.
  • The graft-versus-leukemia effect using matched unrelated donors is not superior to HLA-identical siblings for hematopoietic stem cell transplantation.
  • Which donor or graft source should you choose for the strongest GVL? Is there really any difference.
  • Proceedings from the National Cancer Institute’s Second International Workshop on the Biology, Prevention, and Treatment of Relapse after hematopoietic stem cell transplantation: Part I. Biology of relapse after transplantation.

 

DONOR LYMPHOCYTE INFUSIONS

  • Adoptive immunotherapy evaluating escalating doses of donor leukocytes for relapse of chronic myeloid leukemia after bone marrow transplantation: separation of graft-versus-leukemia responses from graft-versus-host disease.
  • Role of donor lymphocyte infusions in relapsed hematological malignancies after stem cell transplantation revisited.
  • Proceedings from the National Cancer Institute’s second international workshop on the biology, prevention and treatment of relapse after hematopoietic stem cell transplantation: Part III. Prevention and treatment of relapse after allogeneic transplantation.

 

TRANSPLANT-ASSOCIATED COMPLICATIONS.
VENO-OCCLUSIVE DISEASE (VOD) / SINUSOIDAL OBSTRUCTIVE SYNDROME (SOS)

 

INFECTIOUS COMPLICATIONS OF HEMATOPOIETIC STEM CELL TRANSPLANT

Fungal Infections

  • Randomized, double-blind trial of fluconazole versus voriconazole for prevention of invasive fungal infection after allogeneic hematopoietic cell transplantation
  • Prospective surveillance for invasive fungal infections in hematopoietic stem cell transplant recipients, 2001-2006: overview of the Transplant Associated Infection Surveillance Network (TRANSNET) Database.
  • Posaconazole or fluconazole for prophylaxis in severe graft-versus-host disease

Adenovirus

  • Invasive adenoviral infections in T-cell-depleted allogeneic hematopoietic stem cell transplantation: high mortality in the era of cidofovir.
  • Multicenter study of banked third-party virus-specific T cells to treat severe viral infections after hematopoietic stem cell transplantation.
  • European guidelines for diagnosis and treatment of adenovirus infection in leukemia and stem cell transplantation: summary of ECIL-4 (2011).
  • How I treat adenovirus in hematopoietic stem cell transplant recipients.

Human Herpes Virus – 6 (HHV-6)

 

EBV Post-Transplant Lymphoproliferative Disorder (EBV-PTLD)

  • Preemptive diagnosis and treatment of Epstein-Barr virus-associated post transplant lymphoproliferative disorder after hematopoietic stem cell transplant: an approach in development.
  • Prevention of Epstein-Barr virus-lymphoproliferative disease by molecular monitoring and preemptive rituximab in high-risk patients after allogeneic stem cell transplantation.
  • How I treat EBV lymphoproliferation.
  • Guidelines for preventing infectious complications among hematopoietic stem cell transplantation recipients: a global perspective.

 

Cytomegalovirus (CMV)

  • Forscarnet vs. ganciclovir for cytomegalovirus (CMV) antigenemia after allogeneic hemopoietic stem cell transplantation (HSCT): a randomised study.
  • Oral valganciclovir versus ganciclovir as delayed pre-emptive therapy for patients after allogeneic hematopoietic stem cell transplant: a pilot trial (04-0274) and review of the literature.
  • Cytomegalovirus in hematopoietic stem cell transplant recipients
  • How we treat cytomegalovirus in hematopoietic cell transplant recipients.
  • Cytomegalovirus in hematopoietic stem cell transplant recipients: Current status, known challenges, and future strategies.
  • Donor cytomegalovirus status influences the outcome of allogeneic stem cell transplant: a study by the European group for blood and marrow transplantation.
  • Letermovir for cytomegalovirus prophylaxis in hematopoietic-cell transplantation.
  • CMX001 to prevent cytomegalovirus disease in hematopoietic-cell transplantation.

 

Herpes Simplex Virus (HSV) & Varicella Zoster Virus (VZV)

  • Long-term acyclovir for prevention of varicella zoster virus disease after allogeneic hematopoietic cell transplantation--a randomized double-blind placebo-controlled study.
  • One-year acyclovir prophylaxis for preventing varicella-zoster virus disease after hematopoietic cell transplantation: no evidence of rebound varicella-zoster virus disease after drug discontinuation.
  • Clinical utility of oral valacyclovir compared with oral acyclovir for the prevention of herpes simplex virus mucositis following autologous bone marrow transplantation or stem cell rescue therapy

Respiratory Viral Infections

  • The challenge of respiratory virus infections in hematopoietic cell transplant recipients.
  • Fourth European conference on infections in leukaemia (ECIL-4): guidelines for diagnosis and treatment of human respiratory syncytial virus, parainfluenza virus, metapneumovirus, rhinovirus, and coronavirus.
  • Pretransplantation respiratory syncytial virus infection: impact of a strategy to delay transplantation.
  • Randomized controlled multicenter trial of aerosolized ribavirin for respiratory syncytial virus upper respiratory tract infection in hematopoietic cell transplant recipients.
  • How I treat influenza in patients with hematologic malignancies
  • Parainfluenza virus lower respiratory tract disease after hematopoietic cell transplant: viral detection in the lung predicts outcome.
  • Impact of aerosolized ribavirin on mortality in 280 allogeneic haematopoietic stem cell transplant recipients with respiratory syncytial virus infections.
  • Management of RSV in adult recipients of hematopoietic stem cell transplantation.
  • Human parainfluenza virus infection after hematopoietic stem cell transplantation: risk factors, management, mortality and changes over time.
  • Palivizumab treatment of respiratory syncytial virus infection after allogeneic hematpoietic stem cell transplantation

 

BK Virus

  • Polyomavirus BK infection in blood and marrow transplant recipients
  • BK virus and its role in hematopoietic stem cell transplantation: evolution of a pathogen.
  • Ciprofloxacin decreased polyoma BK virus load in patients who underwent allogeneic hematopoietic stem cell transplantation.
  • Low-dose cidofovir in the treatment of symptomatic BK virus infection in patients undergoing allogeneic stem cell transplantation: a retrospective analysis of an algorithmic approach.

 

Bacterial Infections

  • Hematopoietic stem cell transplantation: An overview of infection risks and epidemiology
  • Guidelines or preventing infectious complications among hematopoietic cell transplantation recipients: A global perspective.
  • Bacterial infections in hematopoietic stem cell transplantation recipients.
  • Clostridium dfficile: deleterious impact on hematopoietic stem cell transplantation.
  • Clinical practice guideline for the use of antimicrobial agents in neutropenic patients with cancer: 2010 update by the Infectious Diseases Society of America.

Vaccinations

  • 2013 IDSA Clinical Practice Guidelines for Vaccination of the Immunocompromised Host.