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

Kahn, Justine M.; Brazauskas, Ruta; Tecca, Heather R.; Bo-Subait, Stephanie; Buchbinder, David; Battiwala, Minoo; Flowers, Mary E.D.; Savani, Bipin N.; Phelan, Rachel; Broglie, Larisa; Abraham, Allistair; Keating, Amy K.; Daly, Andrew; Wirk, Baldeep; George, Biju; Alter, Blanche P.; Üstün, Celalettin; Freytes, César O.; Beitinjaneh, Amer; Duncan, Christine; Copelan, Edward A.; Murthy, Hemant S.; Lazarus, Hillard M.; Auletta, Jeffery J.; Myers, Kasiani C.; Williams, Kirsten M.; Page, Kristin; Vrooman, Lynda M.; Norkin, Maxim; Byrne, Michael; Díaz, Miguel Ángel; Kamani, Naynesh; Bhatt, Neel S.; Rezvani, Andrew R.; Farhadfar, Nosha; Mehta, Parinda A.; Hematti, Peiman; Shaw, Peter J.; Kamble, Rammurti T.; Schears, Raquel M.; Olsson, Richard F.; Hayashi, Robert J.; Gale, Robert Peter; Mayo, Samantha; Chhabra, Saurabh; Rotz, Seth J.; Badawy, Sherif M.; Ganguly, Siddhartha; Pavletić, Steven Z.; Nishihori, Taiga; Prestidge, Tim; Agrawal, Vaibhav; Inamoto, Yoshihiro; Shaw, Bronwen E.; Satwani, Prakash

doi:10.1182/bloodadvances.2019000839

Cited by 21 publications

(28 citation statements)

References 30 publications

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“…One patient had two HPCTs, and the other patient had a very slow autologous recovery after graft failure. This low frequency of pre‐malignant and malignant conditions post transplant is consistent with the current literature 34–36 …”

Section: Discussionsupporting

confidence: 91%

Non‐myeloablative human leukocyte antigen‐matched related donor transplantation in sickle cell disease: outcomes from three independent centres

et al. 2021

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Summary Non‐myeloablative haematopoietic progenitor cell transplantation (HPCT) from matched related donors (MRD) has been increasingly utilized in sickle cell disease (SCD). A total of 122 patients received 300 cGy of total body irradiation (TBI), alemtuzumab, unmanipulated filgrastim‐mobilized peripheral blood HPC and sirolimus. The median follow‐up was four years; median age at HPCT was 29 years. Median neutrophil and platelet engraftment occurred on day 22 and 19 respectively; 41 patients required no platelet transfusions. Overall and sickle‐free survival at one and five years were 93% and 85% respectively. Age, sex, pre‐HPCT sickle complications, ferritin and infused HPC numbers were similar between graft failure and engrafted patients. Mean donor myeloid chimaerism at one and five years post HPCT were 84% and 88%, and CD3 was 48% and 53% respectively. Two patients developed grade 1 and 2 skin graft‐versus‐host disease (GVHD) with no chronic GVHD. Median days of recipients taking immunosuppression were 489; 83% of engrafted patients have discontinued immunosuppression. Haemoglobin, haemolytic parameters and hepatic iron levels improved post HPCT. Pulmonary function testing, hepatic histology and neurovascular imaging remained stable, suggesting cessation of further sickle‐related injury. Fourteen patients had children. In this largest group of adult SCD patients, this regimen was highly efficacious, well‐tolerated despite compromised organ functions pre HPCT, and without clinically significant GVHD.

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Section: Discussionsupporting

confidence: 91%

Non‐myeloablative human leukocyte antigen‐matched related donor transplantation in sickle cell disease: outcomes from three independent centres

et al. 2021

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“…The only curative approach is the replacement of impaired cells by healthy donor hematopoietic stem cells and allogeneic HCT (allo HCT) (108)(109)(110). One of the clinical implications of HCT is the long-term complications that cause chronic and irreversible damage to various organs (111), including the endocrine glands. The major potential causes for long-term complications are conditioning regimens with cytotoxic agents and therapies for GVHD, including glucocorticoids (71).…”

Section: Endocrine and Metabolic Disorders Caused By Hct For Iei Impaired Reproductive Function Caused By Hctmentioning

confidence: 99%

Endocrinopathies in Inborn Errors of Immunity

et al. 2021

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Inborn errors of immunity (IEI), caused by hereditary or genetic defects, are a group of more than 400 disorders, in which the immune system, including lymphocytes, neutrophils, macrophages, and complements, does not function properly. The endocrine system is frequently affected by IEI as an associated clinical feature and a complex network of glands which regulate many important body functions, including growth, reproduction, homeostasis, and energy regulation. Most endocrine disorders associated with IEI are hypofunction which would be treated with supplementation therapy, and early diagnosis and appropriate management are essential for favorable long-term outcomes in patients with IEI. In this review, we aimed to comprehensively summarize and discuss the current understanding on the clinical features and the pathophysiology of endocrine disorders in IEI. This review is composed with three parts. First, we discuss the two major pathophysiology of endocrinopathy in IEI, autoimmune response and direct effects of the responsible genes. Next, the details of each endocrinopathy, such as growth failure, hypothyroidism, hypoparathyroidism, adrenal insufficiency, diabetes mellitus (DM) are specified. We also illustrated potential endocrinopathy due to hematopoietic stem cell transplantation, including hypogonadism and adrenal insufficiency due to glucocorticoid therapy.

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“…In a large registry study by the Center for International Blood and Marrow Transplant Research (CIBM-TR) 181 , Kahn JM et al, 2020 examined the risk of SNs and late mortality in 6028 children and adolescents undergoing allo-HSCT for non-malignant diseases (NMDs) between 1995-2012. Median age was 6 years (interquartile range, 1-11; range, 1 to 20).…”

Section: Secondary Malignancies Following Hsctmentioning

confidence: 99%

Long-Term Follow-Up of Pediatric Patients Undergoing Hematopoietic Stem Cell Transplantation

Garcia

Macedo²,

Pelegrina

et al. 2021

jbmtct

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Hematopoietic stem cell transplantation (HSCT) offers the opportunity for cure to patients with malignant and non-malignant diseases. Given the myriad advances in the past few decades, coupled with the rising numbers of transplants worldwide, the number of long-term survivors, many of whom are free of the disease for which they were transplanted, is constantly increasing. Despite the improved prognosis observed overall, long-term outcome may be undermined by transplant-associated morbidity and mortality. Long-term survivors may present a variety of complications, comprising physical, psychological, social, and economic arenas, with a deep impact on quality of life. Therefore, drawing greater attention to and raising awareness of the potential long-term effects of HSCT is key to providing a tailored approach to pretransplant counseling and to devising appropriate recommendations for post-transplant screening, prevention, and timely treatment of secondary events. In 2020, the Brazilian Group for Pediatric Bone Marrow Transplantation of the Brazilian Society for Blood and Marrow Transplantation and Cellular Therapy (SBTMO) convened a task force to provide updated, evidence-based guidance for the long- term follow-up of pediatric patients undergoing HSCT, the results of which are presented here.

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

Cited by 21 publications

References 30 publications

Non‐myeloablative human leukocyte antigen‐matched related donor transplantation in sickle cell disease: outcomes from three independent centres

Non‐myeloablative human leukocyte antigen‐matched related donor transplantation in sickle cell disease: outcomes from three independent centres

Endocrinopathies in Inborn Errors of Immunity

Long-Term Follow-Up of Pediatric Patients Undergoing Hematopoietic Stem Cell Transplantation

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