Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2022 Nov 26;7(2):bvac183.
doi: 10.1210/jendso/bvac183. eCollection 2022 Dec 15.

Endocrine Sequelae in 157 Pediatric Survivors of Hematopoietic Stem Cell Transplantation (HSCT)

María Güemes  1   2 , Álvaro Martín-Rivada  1   2 , Marta Bascuas Arribas  1 , Eva María Andrés-Esteban  3 , Blanca Molina Angulo  4 , Jesús Pozo Román  1   2   5   6 , Jesús Argente  1   2   5   6   7
Affiliations

Endocrine Sequelae in 157 Pediatric Survivors of Hematopoietic Stem Cell Transplantation (HSCT)

María Güemes et al. J Endocr Soc. .

Abstract

Context: Successful rates of hematopoietic stem cell transplantation (HSCT) face paralleled escalation of late endocrine and metabolic effects.

Objective: This work aimed to characterize these sequelae distinguishing between the underlying pathologies and treatments received.

Methods: A retrospective descriptive study was conducted in 157 children post-HSCT (hematopoietic pathology [N = 106], solid tumors [N = 40], and rare entities [N = 11]) followed at a single endocrine department between 2009 and 2019. Regression analysis was used to ascertain association.

Results: Of all patients, 58.7% presented with at least one endocrine abnormality. Endocrinopathies post HSCT were most frequently developed in lymphoblastic leukemia (60.5% of them), whereas myeloid leukemias had the fewest. A total of 64% of patients presented with primary hypogonadism, 52% short stature, and 20% obesity. Endocrinopathy was associated with older age at HSCT (9.78 years [6.25-12.25] vs 6.78 years [4.06-9.75]) (P < .005), pubertal Tanner stage V (P < .001), chronic graft-vs-host disease (GVHD) (P = .022), and direct gonadal therapy (P = .026). The incidence of endocrinopathies was higher in girls (15% more common; P < .02) and in patients who received radiotherapy (18% higher), steroids (17.4% increase), allogenic HSCT (7% higher), thymoglobulin, or cyclophosphamide. Those on busulfan presented with a 27.5% higher rate of primary hypogonadism (P = .003).

Conclusion: More than half of children surviving HSCT will develop endocrinopathies. Strikingly, obesity has risen to the third most frequent endocrine disruption, mainly due to steroids, and partly adhering to the general population tendency. Lymphoblastic leukemia was the condition with a higher rate of endocrine abnormalities. Female sex, older age at HSCT, pubertal stage, allogenic transplant, radiotherapy, alkylating drugs, and GVHD pose risk factors for endocrine disturbances.

Keywords: endocrinopathies; hematopoietic stem cell transplantation; pediatric.

PubMed Disclaimer

Figures

Figure 1.
Figure 1.
Endocrine abnormalities detected before and after HSCT. BMD, bone mineral density; GH, growth hormone; HSCT, hematopoietic stem cell transplantation; Post-HSCT, endocrine abnormalities detected after transplantation; Pre-HSCT, endocrine abnormalities detected before transplantation; T2DM, type 2 diabetes mellitus.
See this image and copyright information in PMC

References

    1. Hows JM, Passweg JR, Tichelli A, et al. ; IMUST Study Participating Centers; Late Effects Working Party of the European Group for Blood and Marrow Transplantation (EBMT). . Comparison of long-term outcomes after allogeneic hematopoietic stem cell transplantation from matched sibling and unrelated donors. Bone Marrow Transplant. 2006;38(12):799–805. - PubMed
    1. Roziakova L, Mladosievicova B. Endocrine late effects after hematopoietic stem cell transplantation. Oncol Res. 2009;18(11-12):607–615. - PubMed
    1. Gyurkocza B, Sandmaier BM. Conditioning regimens for hematopoietic cell transplantation: one size does not fit all. Blood. 2014;124(3):344–353. - PMC - PubMed
    1. Ruutu T, Gratwohl A, de Witte T, et al. . Prophylaxis and treatment of GVHD: EBMT-ELN working group recommendations for a standardized practice. Bone Marrow Transplant. 2014;49(2):168–173. - PubMed
    1. Shalitin S, Pertman L, Yackobovitch-Gavan M, et al. . Endocrine and metabolic disturbances in survivors of hematopoietic stem cell transplantation in childhood and adolescence. Horm Res Paediatr. 2018;89(2):108–121. - PubMed

LinkOut - more resources