Skip to main content
Erschienen in:

22.02.2022 | Original Article

Differences in longitudinal growth patterns of children and adolescents with transfusion-dependent hemoglobin E/β-thalassemia and those achieving successful hematopoietic stem-cell transplantation

verfasst von: Sansanee Jitpirasakun, Julaporn Pooliam, Charotorn Sriwichakorn, Kleebsabai Sanpakit, Pairunyar Nakavachara

Erschienen in: International Journal of Hematology | Ausgabe 4/2022

Einloggen, um Zugang zu erhalten

Abstract

Background

Short stature is a very common endocrinopathy among children with transfusion-dependent (TD) thalassemia. Hematopoietic stem-cell transplantation (HSCT) is the only effective curative treatment for TD thalassemia. This study aimed to identify and compare the longitudinal growth patterns of children with TD hemoglobin E (Hb E)/β-thalassemia against those of children successfully undergoing HSCT.

Materials and methods

We reviewed the medical records of 39 patients with TD Hb E/β-thalassemia receiving regular blood transfusions, and 39 post-HSCT patients. Longitudinal weight and height Z-scores at each year of age were recorded for TD patients, and longitudinal weight and height Z-scores at each year before and after HSCT were obtained for post-HSCT patients.

Results

The mean weight and height Z-scores of TD patients decreased gradually and were lowest at age 13. However, post-HSCT subjects saw significant improvement in their mean weight and height Z-scores 6 and 3 years after HSCT, respectively, relative to pre-HSCT baseline values.

Conclusions

Longitudinal growth patterns differed between patients successfully undergoing HSCT and children and adolescents with TD Hb E/β-thalassemia. HSCT significantly improved height outcomes of children and adolescents with TD Hb E/β-thalassemia.
Literatur
1.
2.
Zurück zum Zitat Chern JP, Lin KH, Tsai WY, Wang SC, Lu MY, Lin DT, et al. Hypogonadotropic hypogonadism and hematologic phenotype in patients with transfusion-dependent beta-thalassemia. J Pediatr Hematol Oncol. 2003;25(11):880–4.CrossRef Chern JP, Lin KH, Tsai WY, Wang SC, Lu MY, Lin DT, et al. Hypogonadotropic hypogonadism and hematologic phenotype in patients with transfusion-dependent beta-thalassemia. J Pediatr Hematol Oncol. 2003;25(11):880–4.CrossRef
3.
Zurück zum Zitat Chatterjee R, Bajoria R. Critical appraisal of growth retardation and pubertal disturbances in thalassemia. Ann N Y Acad Sci. 2010;1202:100–14.CrossRef Chatterjee R, Bajoria R. Critical appraisal of growth retardation and pubertal disturbances in thalassemia. Ann N Y Acad Sci. 2010;1202:100–14.CrossRef
4.
Zurück zum Zitat Benigno V, Bertelloni S, Baroncelli GI, Bertacca L, Di Peri S, Cuccia L, et al. Effects of thalassemia major on bone mineral density in late adolescence. J Pediatr Endocrinol Metab. 2003;16(Suppl 2):337–42.PubMed Benigno V, Bertelloni S, Baroncelli GI, Bertacca L, Di Peri S, Cuccia L, et al. Effects of thalassemia major on bone mineral density in late adolescence. J Pediatr Endocrinol Metab. 2003;16(Suppl 2):337–42.PubMed
5.
Zurück zum Zitat Shamshirsaz AA, Bekheirnia MR, Kamgar M, Pakbaz Z, Tabatabaie SM, Bouzari N, et al. Bone mineral density in Iranian adolescents and young adults with beta-thalassemia major. Pediatr Hematol Oncol. 2007;24(7):469–79.CrossRef Shamshirsaz AA, Bekheirnia MR, Kamgar M, Pakbaz Z, Tabatabaie SM, Bouzari N, et al. Bone mineral density in Iranian adolescents and young adults with beta-thalassemia major. Pediatr Hematol Oncol. 2007;24(7):469–79.CrossRef
6.
Zurück zum Zitat Nakavachara P, Viprakasit V. Children with hemoglobin E/β-thalassemia have a high risk of being vitamin D deficient even if they get abundant sun exposure: a study from Thailand. Pediatr Blood Cancer. 2013;60(10):1683–8.CrossRef Nakavachara P, Viprakasit V. Children with hemoglobin E/β-thalassemia have a high risk of being vitamin D deficient even if they get abundant sun exposure: a study from Thailand. Pediatr Blood Cancer. 2013;60(10):1683–8.CrossRef
7.
Zurück zum Zitat Nakavachara P, Viprakasit V. Adrenal insufficiency is prevalent in HbE/beta-thalassaemia paediatric patients irrespective of their clinical severity and transfusion requirement. Clin Endocrinol (Oxf). 2013;79(6):776–83.CrossRef Nakavachara P, Viprakasit V. Adrenal insufficiency is prevalent in HbE/beta-thalassaemia paediatric patients irrespective of their clinical severity and transfusion requirement. Clin Endocrinol (Oxf). 2013;79(6):776–83.CrossRef
8.
Zurück zum Zitat De Sanctis V, Galimberti M, Lucarelli G, Angelucci E, Ughi M, Baronciani D, et al. Pubertal development in thalassaemic patients after allogenic bone marrow transplantation. Eur J Pediatr. 1993;152(12):993–7.CrossRef De Sanctis V, Galimberti M, Lucarelli G, Angelucci E, Ughi M, Baronciani D, et al. Pubertal development in thalassaemic patients after allogenic bone marrow transplantation. Eur J Pediatr. 1993;152(12):993–7.CrossRef
9.
Zurück zum Zitat Rahal I, Galambrun C, Bertrand Y, Garnier N, Paillard C, Frange P, et al. Late effects after hematopoietic stem cell transplantation for beta-thalassemia major: the French national experience. Haematologica. 2018;103(7):1143–9.CrossRef Rahal I, Galambrun C, Bertrand Y, Garnier N, Paillard C, Frange P, et al. Late effects after hematopoietic stem cell transplantation for beta-thalassemia major: the French national experience. Haematologica. 2018;103(7):1143–9.CrossRef
10.
Zurück zum Zitat Leung TF, Hung ECW, Lam CWK, Li CK, Chu Y, Chik KW, et al. Bone mineral density in children with thalassaemia major: determining factors and effects of bone marrow transplantation. Bone Marrow Transplant. 2005;36(4):331–6.CrossRef Leung TF, Hung ECW, Lam CWK, Li CK, Chu Y, Chik KW, et al. Bone mineral density in children with thalassaemia major: determining factors and effects of bone marrow transplantation. Bone Marrow Transplant. 2005;36(4):331–6.CrossRef
11.
Zurück zum Zitat Nokeaingtong K, Charoenkwan P, Silvilairat S, Saekho S, Pongprot Y, Dejkhamron P. A longitudinal study of growth and relation with anemia and iron overload in pediatric patients with transfusion-dependent thalassemia. J Pediatr Hematol Oncol. 2016;38(6):457–62.CrossRef Nokeaingtong K, Charoenkwan P, Silvilairat S, Saekho S, Pongprot Y, Dejkhamron P. A longitudinal study of growth and relation with anemia and iron overload in pediatric patients with transfusion-dependent thalassemia. J Pediatr Hematol Oncol. 2016;38(6):457–62.CrossRef
12.
Zurück zum Zitat Tan KA, Lum SH, Yahya A, Krishnan S, Jalaludin MY, Lee WS. Prevalence of growth and endocrine disorders in Malaysian children with transfusion-dependent thalassaemia. Singapore Med J. 2019;60(6):303–8.CrossRef Tan KA, Lum SH, Yahya A, Krishnan S, Jalaludin MY, Lee WS. Prevalence of growth and endocrine disorders in Malaysian children with transfusion-dependent thalassaemia. Singapore Med J. 2019;60(6):303–8.CrossRef
13.
Zurück zum Zitat Saka N, Sükür M, Bundak R, Anak S, Neyzi O, Gedikoğlu G. Growth and puberty in thalassemia major. J Pediatr Endocrinol Metab. 1995;8(3):181–6.CrossRef Saka N, Sükür M, Bundak R, Anak S, Neyzi O, Gedikoğlu G. Growth and puberty in thalassemia major. J Pediatr Endocrinol Metab. 1995;8(3):181–6.CrossRef
14.
Zurück zum Zitat Rathaur VK, Imran A, Pathania M. Growth pattern in thalassemic children and their correlation with serum ferritin. J Family Med Prim Care. 2020;9(2):1166–9.CrossRef Rathaur VK, Imran A, Pathania M. Growth pattern in thalassemic children and their correlation with serum ferritin. J Family Med Prim Care. 2020;9(2):1166–9.CrossRef
15.
Zurück zum Zitat Shalitin S, Carmi D, Weintrob N, Phillip M, Miskin H, Kornreich L, et al. Serum ferritin level as a predictor of impaired growth and puberty in thalassemia major patients. Eur J Haematol. 2005;74(2):93–100.CrossRef Shalitin S, Carmi D, Weintrob N, Phillip M, Miskin H, Kornreich L, et al. Serum ferritin level as a predictor of impaired growth and puberty in thalassemia major patients. Eur J Haematol. 2005;74(2):93–100.CrossRef
16.
Zurück zum Zitat Shamshirsaz AA, Bekheirnia MR, Kamgar M, Pourzahedgilani N, Bouzari N, Habibzadeh M, et al. Metabolic and endocrinologic complications in beta-thalassemia major: a multicenter study in Tehran. BMC Endocr Disord. 2003;3(1):4.CrossRef Shamshirsaz AA, Bekheirnia MR, Kamgar M, Pourzahedgilani N, Bouzari N, Habibzadeh M, et al. Metabolic and endocrinologic complications in beta-thalassemia major: a multicenter study in Tehran. BMC Endocr Disord. 2003;3(1):4.CrossRef
17.
Zurück zum Zitat Dhouib NG, Ben Khaled M, Ouederni M, Besbes H, Kouki R, Mellouli F, et al. Growth and endocrine function in Tunisian thalassemia major patients. Mediterr J Hematol Infect Dis. 2018;10(1): e2018031.CrossRef Dhouib NG, Ben Khaled M, Ouederni M, Besbes H, Kouki R, Mellouli F, et al. Growth and endocrine function in Tunisian thalassemia major patients. Mediterr J Hematol Infect Dis. 2018;10(1): e2018031.CrossRef
18.
Zurück zum Zitat Hashemi AS, Ghilian R, Golestan M, Akhavan Ghalibaf M, Zare Z, Dehghani MA. The study of growth in thalassemic patients and its correlation with serum ferritin level. Iran J PedHematol Oncol. 2011;1(4):147–51. Hashemi AS, Ghilian R, Golestan M, Akhavan Ghalibaf M, Zare Z, Dehghani MA. The study of growth in thalassemic patients and its correlation with serum ferritin level. Iran J PedHematol Oncol. 2011;1(4):147–51.
19.
Zurück zum Zitat De Simone M, Verrotti A, Iughetti L, Palumbo M, Di Bartolomeo P, Olioso P, et al. Final height of thalassemic patients who underwent bone marrow transplantation during childhood. Bone Marrow Transplant. 2001;28(2):201–5.CrossRef De Simone M, Verrotti A, Iughetti L, Palumbo M, Di Bartolomeo P, Olioso P, et al. Final height of thalassemic patients who underwent bone marrow transplantation during childhood. Bone Marrow Transplant. 2001;28(2):201–5.CrossRef
20.
Zurück zum Zitat Aldemir-Kocabas B, Tezcan-Karasu G, Bircan I, Bircan O, Aktas-Samur A, Yesilipek MA. Evaluating the patients with thalassemia major for long-term endocrinological complications after bone marrow transplantation. Pediatr Hematol Oncol. 2014;31(7):616–23.CrossRef Aldemir-Kocabas B, Tezcan-Karasu G, Bircan I, Bircan O, Aktas-Samur A, Yesilipek MA. Evaluating the patients with thalassemia major for long-term endocrinological complications after bone marrow transplantation. Pediatr Hematol Oncol. 2014;31(7):616–23.CrossRef
21.
Zurück zum Zitat See WQ, Tung JY, Cheuk DK, Hwang YY, Ip JK, Cheung PT, et al. Endocrine complications in patients with transfusion-dependent thalassaemia after haemopoietic stem cell transplantation. Bone Marrow Transplant. 2018;53(3):356–60.CrossRef See WQ, Tung JY, Cheuk DK, Hwang YY, Ip JK, Cheung PT, et al. Endocrine complications in patients with transfusion-dependent thalassaemia after haemopoietic stem cell transplantation. Bone Marrow Transplant. 2018;53(3):356–60.CrossRef
22.
Zurück zum Zitat Li CK, Chik KW, Wong GW, Cheng PS, Lee V, Shing MM. Growth and endocrine function following bone marrow transplantation for thalassemia major. Pediatr Hematol Oncol. 2004;21(5):411–9.CrossRef Li CK, Chik KW, Wong GW, Cheng PS, Lee V, Shing MM. Growth and endocrine function following bone marrow transplantation for thalassemia major. Pediatr Hematol Oncol. 2004;21(5):411–9.CrossRef
23.
Zurück zum Zitat Olivieri NF, Pakbaz Z, Vichinsky E. HbE/β-thalassemia: basis of marked clinical diversity. Hematol Oncol Clin North Am. 2010;24(6):1055–70.CrossRef Olivieri NF, Pakbaz Z, Vichinsky E. HbE/β-thalassemia: basis of marked clinical diversity. Hematol Oncol Clin North Am. 2010;24(6):1055–70.CrossRef
24.
Zurück zum Zitat Porter J, Viprakasit V, Kattamis A. Iron overload and chelation. In: Cappellini MD, Cohen A, Porter J, Taher A, Viprakasit V, editors. Guidelines for the management of transfusion dependent thalassaemia (TDT). 3rd ed. Nicosia: Thalassaemia International Federation; 2014. p. 42–97. Porter J, Viprakasit V, Kattamis A. Iron overload and chelation. In: Cappellini MD, Cohen A, Porter J, Taher A, Viprakasit V, editors. Guidelines for the management of transfusion dependent thalassaemia (TDT). 3rd ed. Nicosia: Thalassaemia International Federation; 2014. p. 42–97.
25.
Zurück zum Zitat Ekwattanakit S, Nakavachara P, Viprakasit V, Phil D. An excel-based work-sheet program for a rapid calculation of weight-for-age and height-for-age Z-scores in a pediatric Southeast Asian population. Southeast Asian J Trop Med Publ Health. 2018;48:183–91. Ekwattanakit S, Nakavachara P, Viprakasit V, Phil D. An excel-based work-sheet program for a rapid calculation of weight-for-age and height-for-age Z-scores in a pediatric Southeast Asian population. Southeast Asian J Trop Med Publ Health. 2018;48:183–91.
26.
Zurück zum Zitat Collett-Solberg PF, Ambler G, Backeljauw PF, Bidlingmaier M, Biller BMK, Boguszewski MCS, et al. Diagnosis, genetics, and therapy of short stature in children: a growth hormone research society international perspective. Horm Res Paediatr. 2019;92(1):1–14.CrossRef Collett-Solberg PF, Ambler G, Backeljauw PF, Bidlingmaier M, Biller BMK, Boguszewski MCS, et al. Diagnosis, genetics, and therapy of short stature in children: a growth hormone research society international perspective. Horm Res Paediatr. 2019;92(1):1–14.CrossRef
27.
Zurück zum Zitat Anurathapan U, Pakakasama S, Mekjaruskul P, Sirachainan N, Songdej D, Chuansumrit A, et al. Outcomes of thalassemia patients undergoing hematopoietic stem cell transplantation by using a standard myeloablative versus a novel reduced-toxicity conditioning regimen according to a new risk stratification. Biol Blood Marrow Transplant. 2014;20(12):2066–71.CrossRef Anurathapan U, Pakakasama S, Mekjaruskul P, Sirachainan N, Songdej D, Chuansumrit A, et al. Outcomes of thalassemia patients undergoing hematopoietic stem cell transplantation by using a standard myeloablative versus a novel reduced-toxicity conditioning regimen according to a new risk stratification. Biol Blood Marrow Transplant. 2014;20(12):2066–71.CrossRef
28.
Zurück zum Zitat Glucksberg H, Storb R, Fefer A, Buckner CD, Neiman PE, Clift RA, et al. Clinical manifestations of graft-versus-host disease in human recipients of marrow from HL-A-matched sibling donors. Transplantation. 1974;18(4):295–304.CrossRef Glucksberg H, Storb R, Fefer A, Buckner CD, Neiman PE, Clift RA, et al. Clinical manifestations of graft-versus-host disease in human recipients of marrow from HL-A-matched sibling donors. Transplantation. 1974;18(4):295–304.CrossRef
29.
Zurück zum Zitat Lee SJ, Flowers ME. Recognizing and managing chronic graft-versus-host disease. Hematology Am Soc Hematol Educ Program; 2008. p. 134–41. Lee SJ, Flowers ME. Recognizing and managing chronic graft-versus-host disease. Hematology Am Soc Hematol Educ Program; 2008. p. 134–41.
30.
Zurück zum Zitat Chan YL, Li CK, Pang LM, Chik KW. Desferrioxamine-induced long bone changes in thalassaemic patients—radiographic features, prevalence and relations with growth. Clin Radiol. 2000;55(8):610–4.CrossRef Chan YL, Li CK, Pang LM, Chik KW. Desferrioxamine-induced long bone changes in thalassaemic patients—radiographic features, prevalence and relations with growth. Clin Radiol. 2000;55(8):610–4.CrossRef
31.
Zurück zum Zitat Caruso-Nicoletti M, De Sanctis V, Capra M, Cardinale G, Cuccia L, Di Gregorio F, et al. Short stature and body proportion in thalassaemia. J Pediatr Endocrinol Metab. 1998;11(Suppl 3):811–6.PubMed Caruso-Nicoletti M, De Sanctis V, Capra M, Cardinale G, Cuccia L, Di Gregorio F, et al. Short stature and body proportion in thalassaemia. J Pediatr Endocrinol Metab. 1998;11(Suppl 3):811–6.PubMed
32.
Zurück zum Zitat Soliman A, ElZalabany M, Amer M, Ansari B. Growth and pubertal development in transfusion-dependent children and adolescents with thalassaemia major and sickle cell disease: a comparative study. J Trop Pediatr. 1999;45(1):23–30.CrossRef Soliman A, ElZalabany M, Amer M, Ansari B. Growth and pubertal development in transfusion-dependent children and adolescents with thalassaemia major and sickle cell disease: a comparative study. J Trop Pediatr. 1999;45(1):23–30.CrossRef
33.
Zurück zum Zitat Kanbour I, Chandra P, Soliman A, De Sanctis V, Nashwan A, Abusamaan S, et al. Severe liver iron concentrations (LIC) in 24 patients with β-thalassemia major: correlations with serum ferritin, liver enzymes and endocrine complications. Mediterr J Hematol Infect Dis. 2018;10(1): e2018062.PubMedPubMedCentral Kanbour I, Chandra P, Soliman A, De Sanctis V, Nashwan A, Abusamaan S, et al. Severe liver iron concentrations (LIC) in 24 patients with β-thalassemia major: correlations with serum ferritin, liver enzymes and endocrine complications. Mediterr J Hematol Infect Dis. 2018;10(1): e2018062.PubMedPubMedCentral
34.
Zurück zum Zitat Wang YCH. Use of percentiles and Z-scores in anthropometry. In: Preedy VR, editor. Handbook of anthropometry: physical measures of human form in health and disease. New York: Springer; 2012. p. 29–48.CrossRef Wang YCH. Use of percentiles and Z-scores in anthropometry. In: Preedy VR, editor. Handbook of anthropometry: physical measures of human form in health and disease. New York: Springer; 2012. p. 29–48.CrossRef
35.
Zurück zum Zitat Chaudhury S, Ayas M, Rosen C, Ma M, Viqaruddin M, Parikh S, et al. A multicenter retrospective analysis stressing the importance of long-term follow-up after hematopoietic cell transplantation for beta-thalassemia. Biol Blood Marrow Transplant. 2017;23(10):1695–700.CrossRef Chaudhury S, Ayas M, Rosen C, Ma M, Viqaruddin M, Parikh S, et al. A multicenter retrospective analysis stressing the importance of long-term follow-up after hematopoietic cell transplantation for beta-thalassemia. Biol Blood Marrow Transplant. 2017;23(10):1695–700.CrossRef
36.
Zurück zum Zitat Van Metre TE Jr, Niermann WA, Rosen LJ. A comparison of the growth suppressive effect of cortisone, prednisone, and other adrenal cortical hormones. J Allergy. 1960;31:531–42.CrossRef Van Metre TE Jr, Niermann WA, Rosen LJ. A comparison of the growth suppressive effect of cortisone, prednisone, and other adrenal cortical hormones. J Allergy. 1960;31:531–42.CrossRef
Metadaten
Titel
Differences in longitudinal growth patterns of children and adolescents with transfusion-dependent hemoglobin E/β-thalassemia and those achieving successful hematopoietic stem-cell transplantation
verfasst von
Sansanee Jitpirasakun
Julaporn Pooliam
Charotorn Sriwichakorn
Kleebsabai Sanpakit
Pairunyar Nakavachara
Publikationsdatum
22.02.2022
Verlag
Springer Singapore
Erschienen in
International Journal of Hematology / Ausgabe 4/2022
Print ISSN: 0925-5710
Elektronische ISSN: 1865-3774
DOI
https://doi.org/10.1007/s12185-021-03279-4

Neu im Fachgebiet Onkologie

KI erlaubt neue Einsichten in die Krebsprognose

Forschende sind in komplexen Real-World-Daten des Westdeutschen Tumorzentrums auf bisher weniger beachtete Zusammenhänge von bestimmten Risikomarkern mit der Prognose gestoßen. Möglich gemacht hat’s ein Verfahren namens „erklärbare KI“, mit dem sich hunderte Tumor- und Patientenmerkmale gleichzeitig betrachten lassen.

Körperlich fitte Krebskranke leben länger

Krebspatienten, auch und vor allem solche in fortgeschrittenen Stadien, profitieren offenbar von guter körperlicher Verfassung. Hohe Muskelkraft und kardiorespiratorische Fitness sind laut Ergebnissen einer Metaanalyse mit geringerer Mortalität assoziiert.

Kein (großer) Schutz vor Kolorektalkarzinom-Rezidiven durch ASS

Die erste Phase-3-Studie zum Nutzen von ASS in der adjuvanten Therapie des kolorektalen Karzinoms ist negativ verlaufen. Das abschließende Urteil über eine Sekundärprävention mit ASS ist trotzdem noch nicht gefallen.

Frühe CLL-Therapie: BTK-Hemmer verlängert EFS und PFS, aber nicht OS

Auch nach sechs Jahren ergibt sich kein Überlebensvorteil einer Therapie mit dem BTK-Hemmer Ibrutinib für Menschen mit frühem CLL-Stadium und erhöhtem Progressionsrisiko. Die Progressionsrate wird mit der Behandlung jedoch um über 80% gesenkt.

Update Onkologie

Bestellen Sie unseren Fach-Newsletter und bleiben Sie gut informiert.