Late Effects Screening Guidelines after Hematopoietic Cell Transplantation (HCT) for Hemoglobinopathy: Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric HCT

Shenoy, Shalini; Gaziev, Javid; Angelucci, Emanuele; King, Allison A.; Bhatia, Monica; Smith, Angela R.; Bresters, Dorine; Haight, Anne E.; Duncan, Christine; Fuente, Josu de la; Dietz, Andrew C.; Baker, K. Scott; Pulsipher, Michael A.; Walters, Mark C.

doi:10.1016/j.bbmt.2018.04.002

Cited by 44 publications

(45 citation statements)

References 66 publications

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“…Here we report contemporary data on a diverse patient population to both inform decision making and contribute to consensus guidelines about posttransplantation follow-up surveillance in children and adolescents receiving transplants for NMDs. 5 Patients and methods…”

Section: Introductionmentioning

confidence: 99%

Subsequent neoplasms and late mortality in children undergoing allogeneic transplantation for nonmalignant diseases

Kahn

Brazauskas

Tecca³

et al. 2020

Blood Advances

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We examined the risk of subsequent neoplasms (SNs) and late mortality in children and adolescents undergoing allogeneic hematopoietic cell transplantation (HCT) for nonmalignant diseases (NMDs). We included 6028 patients (median age, 6 years; interquartile range, 1-11; range, <1 to 20) from the Center for International Blood and Marrow Transplant Research (1995-2012) registry. Standardized mortality ratios (SMRs) in 2-year survivors and standardized incidence ratios (SIRs) were calculated to compare mortality and SN rates with expected rates in the general population. Median follow-up of survivors was 7.8 years. Diagnoses included severe aplastic anemia (SAA; 24%), Fanconi anemia (FA; 10%), other marrow failure (6%), hemoglobinopathy (15%), immunodeficiency (23%), and metabolic/leukodystrophy syndrome (22%). Ten-year survival was 93% (95% confidence interval [95% CI], 92% to 94%; SMR, 4.2; 95% CI, 3.7-4.8). Seventy-one patients developed SNs (1.2%). Incidence was highest in FA (5.5%), SAA (1.1%), and other marrow failure syndromes (1.7%); for other NMDs, incidence was <1%. Hematologic (27%), oropharyngeal (25%), and skin cancers (13%) were most common. Leukemia risk was highest in the first 5 years posttransplantation; oropharyngeal, skin, liver, and thyroid tumors primarily occurred after 5 years. Despite a low number of SNs, patients had an 11-fold increased SN risk (SIR, 11; 95% CI, 8.9-13.9) compared with the general population. We report excellent long-term survival and low SN incidence in an international cohort of children undergoing HCT for NMDs. The risk of SN development was highest in patients with FA and marrow failure syndromes, highlighting the need for long-term posttransplantation surveillance in this population.

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Section: Introductionmentioning

confidence: 99%

Subsequent neoplasms and late mortality in children undergoing allogeneic transplantation for nonmalignant diseases

Kahn

Brazauskas

Tecca³

et al. 2020

Blood Advances

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“…This enhances serial longterm follow-up of neurocognition as providers and patients seek to assess SCD-related complications to plan interventions and develop eligibility criteria for curative measures. Additional effort during clinical trial development, uniformity in comparative testing between protocols [25], and investment in registries to track patients receiving longer-term supportive care and curative therapies terms are immensely important to assess the value of therapeutic interventions for SCD.…”

Section: Discussionmentioning

confidence: 99%

Functional and Radiologic Assessment of the Brain after Reduced-Intensity Unrelated Donor Transplantation for Severe Sickle Cell Disease: Blood and Marrow Transplant Clinical Trials Network Study 0601

King

McKinstry

et al. 2019

Biology of Blood and Marrow Transplantation

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Stroke and cognitive decline are hallmarks of sickle cell disease (SCD). The natural history of SCD predicts progressive loss of 1 IQ point per year attributable to disease-related pathology. Hematopoietic cell transplantation (HCT) is curative by reverting to donor-derived erythropoiesis, but evidence that HCT can positively influence diseaseinduced cognitive decline is lacking. The Sickle Cell Unrelated Transplant Trial prospectively evaluated cognition and brain magnetic resonance imaging (MRI) findings at 2 years after reduced-intensity conditioning followed by unrelated donor HCT. Thirteen study participants completed pre-HCT and post-HCT assessments of intelligence. The mean age of participants was 12.5 § 3.3 years (range, 6.7 to 17.4 years). Eleven of the 13 recipients completed imaging studies at baseline and post-HCT. Seven had overt stroke pre-HCT, and 1 had an elevated transcranial Doppler velocity with abnormal MRI. The mean Full-Scale IQ was stable: 90.9 § 13 at baseline and 91.2 § 13 post-HCT. The mean Performance IQ was 89.9 § 13 at baseline versus 90.9 § 13 post-HCT, and mean Verbal IQ was 93.4 § 13 at baseline versus 93.2 § 13 post-HCT, respectively. Six recipients had stable MRI; 2 showed resolution of all areas of infarction. Three had additional infarcts post-HCT noted at the 2-year time point. This is the first report describing stabilization of IQ and central nervous system outcomes after unrelated donor HCT despite previous central nervous system morbidity and post-HCT posterior reversible encephalopathy syndrome. These preliminary results post-HCT suggest that HCT may stabilize the cognitive decline of SCD and should continue to be followed over the long term.

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“…Comprehensive long‐term data regarding outcomes of transplantation including complications are lacking with only single‐center and small‐cohort data available 34 . Long‐term follow‐up guidelines have recently been published that should help streamline organ evaluation, and research in this area would be valuable to clinicians and families when considering transplantation 35 …”

Section: Discussionmentioning

confidence: 99%

Hematopoietic stem cell transplant referral patterns for children with sickle cell disease vary among pediatric hematologist/oncologists’ practice focus: A Sickle Cell Transplant Advocacy and Research Alliance (STAR) study

Meier

Abraham

Ngwube

et al. 2021

Pediatric Blood & Cancer

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Background: Hematopoietic stem cell transplantation (HSCT) provides a curative therapy for children severely affected by sickle cell disease (SCD). Rejection-free survival after matched sibling donor (MSD) HSCT is very high, but adoption of HSCT as a curative SCD therapy has been slow. In this study, we assess providers' perceptions about MSD HSCT for children with variable SCD severity, and determine the influence of provider characteristics on HSCT referrals. Procedure: After our Institutional Review Board deemed the study exempt, American Society of Pediatric Hematology/Oncology Clinical Forum listserv subscribers and American Society of Hematology members who self-identified as pediatric hematologists/oncologists (PHO) were emailed a survey. Analysis was performed to describe and evaluate correlations between participant demographics (including practice focus within PHO) and likelihood of HSCT referral for each scenario. Results: Spearman's rank correlation analysis did not reveal any significant relationship between demographic characteristics except practice focus and likelihood to refer to

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Late Effects Screening Guidelines after Hematopoietic Cell Transplantation (HCT) for Hemoglobinopathy: Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric HCT

Cited by 44 publications

References 66 publications

Subsequent neoplasms and late mortality in children undergoing allogeneic transplantation for nonmalignant diseases

Subsequent neoplasms and late mortality in children undergoing allogeneic transplantation for nonmalignant diseases

Functional and Radiologic Assessment of the Brain after Reduced-Intensity Unrelated Donor Transplantation for Severe Sickle Cell Disease: Blood and Marrow Transplant Clinical Trials Network Study 0601

Hematopoietic stem cell transplant referral patterns for children with sickle cell disease vary among pediatric hematologist/oncologists’ practice focus: A Sickle Cell Transplant Advocacy and Research Alliance (STAR) study

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