National Cancer Institute, National Heart, Lung and Blood Institute/Pediatric Blood and Marrow Transplantation Consortium First International Consensus Conference on Late Effects after Pediatric Hematopoietic Cell Transplantation: The Need for Pediatric-Specific Long-Term Follow-up Guidelines

Pulsipher, Michael A.; Skinner, Roderick; McDonald, George B.; Hingorani, Sangeeta; Armenian, Saro H.; Cooke, Kenneth R.; Gracia, Clarisa R.; Petryk, Anna; Bhatia, Smita; Bunin, Nancy; Nieder, Michael L.; Dvorak, Christopher C.; Sung, Lillian; Sanders, Jean E.; Kurtzberg, Joanne; Baker, K. Scott

doi:10.1016/j.bbmt.2012.01.003

Cited by 78 publications

(55 citation statements)

References 92 publications

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“…9,10,15 More has also been learned regarding the late effects of transplant conditioning. [16][17][18] A gradual decrease in ablative TBI-based transplants and an increase in IV-BU-based transplants have occurred without supportive prospective data comparing the approaches.…”

Section: Discussionmentioning

confidence: 99%

Prospective cohort study comparing intravenous busulfan to total body irradiation in hematopoietic cell transplantation

Bredeson

Le‐Rademacher

Kato

et al. 2013

Blood

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136

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• Compared with TBI, IV-BU resulted in superior survival with no increased risk for relapse or TRM.• The results support the use of myeloablative IV-BU vs TBIbased conditioning regimens for treatment of myeloid malignancies.We conducted a prospective cohort study testing the noninferiority of survival of ablative intravenous busulfan (IV-BU) vs ablative total body irradiation (TBI)-based regimens in myeloid malignancies. A total of 1483 patients undergoing transplantation for myeloid malignancies (IV-BU, N 5 1025; TBI, N 5 458) were enrolled. Cohorts were similar with respect to age, gender, race, performance score, disease, and disease stage at transplantation. Most patients had acute myeloid leukemia (68% IV-BU, 78% TBI). Grafts were primarily peripheral blood (77%) from HLA-matched siblings (40%) or well-matched unrelated donors (48%). Two-year probabilities of survival (95% confidence interval [CI]), were 56% (95% CI, 53%-60%) and 48% (95% CI, 43%-54%, P 5 .019) for IV-BU (relative risk, 0.82; 95% CI, 0.68-0.98, P 5 .03) and TBI, respectively. Corresponding incidences of transplantrelated mortality (TRM) were 18% (95% CI, 16%-21%) and 19% (95% CI, 15%-23%, P 5 .75) and disease progression were 34% (95% CI, 31%-37%) and 39% (95% CI, 34%-44%, P 5 .08).The incidence of hepatic veno-occlusive disease (VOD) was 5% for IV-BU and 1% with TBI (P < .001). There were no differences in progression-free survival and graft-versus-host disease. Compared with TBI, IV-BU resulted in superior survival with no increased risk for relapse or TRM. These results support the use of myeloablative IV-BU vs TBI-based conditioning regimens for treatment of myeloid malignancies. (Blood. 2013;122(24):3871-3878)

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Section: Discussionmentioning

confidence: 99%

Prospective cohort study comparing intravenous busulfan to total body irradiation in hematopoietic cell transplantation

Bredeson

Le‐Rademacher

Kato

et al. 2013

Blood

Self Cite

136

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“…In addition to the consensus guidelines described above, a framework for late effects screening and management guidelines with a specific pediatric HCT focus has been recently proposed. 20 Although there are many similarities, the latter take into account pre-and post-transplant exposures and late complications that are particularly relevant for pediatric HCT survivors. The Children's Oncology Group has also developed followup guidelines for pediatric cancer survivors that have sections relevant for HCT survivors (available at www.survivorshipguidelines.org).…”

Section: Guidelines For Long-term Followup Of Hct Recipientsmentioning

confidence: 99%

Surviving the cure: long term followup of hematopoietic cell transplant recipients

Majhail

Rizzo

2013

Bone Marrow Transplant

132

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Advances in hematopoietic cell transplantation (HCT) have led to an increasing number of transplant survivors. However, long-term survival is challenged by late relapse, late complications and late non-relapse mortality, and HCT survivors need continued lifelong surveillance for screening, early detection and timely treatment of late complications such as secondary cancers, late infections and organ toxicity. Guidelines for screening and preventive practices for pediatric and adult survivors of autologous and allogeneic HCT were updated and published in 2012. However, several barriers to the care of HCT survivors and routine utilization of these guidelines in clinical practice exist. Examples include paucity of and challenges to conducting prospective randomized trials for screening and prevention of late complications, lack of resources to manage late effects at the level of transplant centers and community health care providers, and inadequate tools to facilitate care and followup of HCT survivors. We summarize the longterm followup guidelines in this review, discuss ways that providers can integrate and utilize them for the care of their patients, and identify areas for research that can inform and increase the utilization of screening and prevention guidelines in clinical practice. Keywords: hematopoietic cell transplantation; long-term survival; late complications; screening; preventive practices INTRODUCTION Several advances in transplantation techniques and supportive care practices over the last four decades have improved long-term survival of patients undergoing autologous or allogeneic hematopoietic cell transplantation (HCT). As patients survive longer, there is an increasing recognition that pre-, peri-and posttransplant exposures have the potential to compromise life expectancy and can contribute to development of late complications. These complications can cause substantial morbidity and mortality and can impair quality of life among HCT survivors. This review will summarize the contemporary literature on long-term survival after autologous and allogeneic HCT and the recently updated guidelines for long-term followup of HCT survivors. Bone Marrow Transplantation

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“…9 The National Cancer Institute, National Heart, Lung and Blood Institute and Pediatric Blood and Marrow Transplantation Consortium have recently published the first International consensus conference report for pediatric-specific long-term follow-up guidelines following SCT. 10 In that report, there is an acknowledgment that early recognition and treatment of pulmonary conditions after SCT is important for successful outcome, and thus increased surveillance for lung dysfunction by serial PFTs for the first 2 years after SCT should be considered whenever feasible. Recommendations from the Joint Transplant Society (CIBMTR, ASBMT, EBMT, APBMT, BMTSANZ, EMBMT and SBTMO) suggest routine clinical evaluation at 6 months and 1 year after SCT and at least yearly thereafter.…”

Section: Introductionmentioning

confidence: 99%

Chest health surveillance utility in the early detection of bronchiolitis obliterans syndrome in children after allo-SCT

Gassas

Craig-Barnes

Dell

et al. 2012

Bone Marrow Transplant

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To prospectively assess whether periodic chest health surveillance is beneficial for the early detection of bronchiolitis obliterans syndrome (BOS) in children after allo-SCT. Children up to 18 years of age receiving allo-SCT from September 2009 to September 2011 were included. Surveillance consisted of the following: a 7-item respiratory system questionnaire of cough, wheeze and shortness of breath; focused physical examination; and pulmonary function test (PFT) conducted before SCT and at 1, 3, 6, 9, 12, 18 and 24 months after SCT. Thirty-nine patients were enrolled. Five children developed BOS at a median time of 192 days (range 94-282). Positive response comparisons between the BOS group vs the non-BOS group were NS for history questionnaire (P ¼ 0.2), heart rate (P ¼ 0.3), respiratory rate (P ¼ 0.3) and oxygen saturation monitoring (P ¼ 0.8). Differences between the two groups for chest auscultation and PFT were statistically significant (P ¼ 0.03 and P ¼ 0.01, respectively). However, chest auscultation in the BOS group was only positive after BOS diagnosis. PFT reduction was evident in the asymptomatic phase (BOS group 33%; non-BOS group 4.5%, P ¼ 0.01). Changes in PFT, but not history/physical examination, allow the early detection of BOS in children after SCT. Our study is limited by the small sample size.

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National Cancer Institute, National Heart, Lung and Blood Institute/Pediatric Blood and Marrow Transplantation Consortium First International Consensus Conference on Late Effects after Pediatric Hematopoietic Cell Transplantation: The Need for Pediatric-Specific Long-Term Follow-up Guidelines

Cited by 78 publications

References 92 publications

Prospective cohort study comparing intravenous busulfan to total body irradiation in hematopoietic cell transplantation

Prospective cohort study comparing intravenous busulfan to total body irradiation in hematopoietic cell transplantation

Surviving the cure: long term followup of hematopoietic cell transplant recipients

Chest health surveillance utility in the early detection of bronchiolitis obliterans syndrome in children after allo-SCT

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