Araştırma Makalesi
BibTex RIS Kaynak Göster

Evaluation of Initial Diagnosis, Clinical-Treatment Features of Children with Acute Immune Thrombocytopenia

Yıl 2025, Cilt: 47 Sayı: 2, 221 - 226, 27.02.2025

Öz

Primary immune thrombocytopenia (ITP), formerly known as idiopathic thrombocytopenic purpura, is the most common cause of thrombocytopenia in children, defined by an isolated low platelet count (<100,000/mm³) without other etiologies. It has an annual occurrence of approximately 2 to 5 per 100,000 children. Acute ITP presents with mucosal bleeding or skin manifestations at diagnosis, with classifications for acute, persistent, and chronic ITP based on the duration of the condition. Most children (70-80%) experience spontaneous recovery within 12 months. Both primary and secondary ITP exhibit immunological abnormalities, yet secondary cases are linked to identifiable etiological factors. The treatment objectives focus on halting active bleeding and preventing future bleeding, as well as enhancing health-related quality of life (HRQoL). Corticosteroids have been the mainstay of ITP treatment since the 1950s, with recent studies showing a trend toward reducing high-dose methylprednisolone (HDMP) use.This retrospective study was conducted in Turkey, analyzing 40 pediatric patients diagnosed with acute ITP who received intravenous methylprednisolone in two groups with varying doses. The results indicated that both treatment regimens achieved similar response rates, and there were no significant differences regarding relapse rates or the development of chronic ITP between the groups. Overall, while initial treatment response rates did not differ significantly, the study emphasizes the need to investigate dosing strategies for managing acute ITP effectively. The findings support the use of a ‘mega dose’ of methylprednisolone for the initial treatment phase, but suggest that a lower dose may be equally effective without compromising patient outcomes.

Etik Beyan

Çalışma Eskişehir Osmangazi Üniversitesi Girişimsel Olmayan Klinik Araştırmalar Etik Kurulu tarafından onaylandı (Karar no: 02, Tarih: 02.01.2024).

Destekleyen Kurum

Yok

Teşekkür

Hayatını tıp alanına adamış değerli hocamız Prof. Dr. Şinasi Özsoylu'yu saygı ve minnetle anıyoruz.

Kaynakça

  • 1. Kochhar M, Neunert C. Immune thrombocytopenia: A review of upfront treatment strategies. Blood Rev. 2021 Sep;49: 100822.
  • 2. Grace RF, Lambert MP. An update on pediatric ITP: differentiating primary ITP, IPD, and PID. Blood. 2022 Aug 11;140(6): 542-555.
  • 3. Rodeghiero F, Stasi R, Gernsheimer T, Michel M, Provan D, Arnold DM, Bussel JB, Cines DB, Chong BH, Cooper N, Godeau B, Lechner K, Mazzucconi MG, McMillan R, Sanz MA, Imbach P, Blanchette V, Kühne T, Ruggeri M, George JN. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood. 2009 Mar 12;113(11): 2386-93.
  • 4. Singh G, Bansal D, Wright NAM. Immune Thrombocytopenia in Children: Consensus and Controversies. Indian J Pediatr. 2020 Feb;87(2): 150-157.
  • 5. Blanchette V, Bolton-Maggs P. Childhood immune thrombocytopenic purpura: diagnosis and management. Pediatr Clin North Am. 2008 Apr;55(2):393-420.
  • 6. Bennett CM, Neunert C, Grace RF, Buchanan G, Imbach P, Vesely SK, Kuhne T. Predictors of remission in children with newly diagnosed immune thrombocytopenia: Data from the Intercontinental Cooperative ITP Study Group Registry II participants. Pediatr Blood Cancer. 2018 Jan;65 (1).
  • 7. Sun S, Urbanus RT, Ten Cate H, de Groot PG, de Laat B, Heemskerk JWM, Roest M. Platelet Activation Mechanisms and Consequences of Immune Thrombocytopenia. Cells. 2021 Dec 1;10(12):3386.
  • 8. Moulinet T, Moussu A, Pierson L, Pagliuca S. The many facets of immune-mediated thrombocytopenia: Principles of immunobiology and immunotherapy. Blood Rev. 2023 Nov 11:101141.
  • 9. Neunert C, Terrell DR, Arnold DM, Buchanan G, Cines DB, Cooper N, Cuker A, Despotovic JM, George JN, Grace RF, Kühne T, Kuter DJ, Lim W, McCrae KR, Pruitt B, Shimanek H, Vesely SK. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019 Dec 10;3(23):3829-3866.
  • 10. Dameshek W, Rubio F Jr, Mahoney JP, Reeves WH, Burgin LA. Treatment of idiopathic thrombocytopenic purpura (ITP) with prednisone. J Am Med Assoc. 1958 Apr 12;166(15):1805-15.
  • 11. Jayabose S, Patel P, Inamdar S, Brilliant R, Mamtani R. Use of intravenous methylprednisolone in acute idiopathic thrombocytopenic purpura. Am J Pediatr Hematol Oncol. 1987 Summer;9(2): 133-5.
  • 12. Ozsoylu S, Irken G, Karabent A. High-dose intravenous methylprednisolone for acute childhood idiopathic thrombocytopenic purpura. Eur J Haematol. 1989 May;42(5): 431-5.
  • 13. Provan D, Arnold DM, Bussel JB, Chong BH, Cooper N, Gernsheimer T, Ghanima W, Godeau B, González-López TJ, Grainger J, Hou M, Kruse C, McDonald V, Michel M, Newland AC, Pavord S, Rodeghiero F, Scully M, Tomiyama Y, Wong RS, Zaja F, Kuter DJ. Updated international consensus report on the investigation and management of primary immune thrombocytopenia. Blood Adv. 2019 Nov 26;3(22):3780-3817.
  • 14. Duru F, Fisgin T, Yarali N, Kara A. Clinical course of children with immune thrombocytopenic purpura treated with intravenous immunoglobulin G or megadose methylprednisolone or observed without therapy. Pediatr Hematol Oncol. 2002 Jun;19 (4):219-25.
  • 15. Erduran E, Aslan Y, Gedik Y, Orhan F. A randomized and comparative study of intravenous immunoglobulin and mega dose methylprednisolone treatments in children with acute idiopathic thrombocytopenic purpura. Turk J Pediatr. 2003 Oct-Dec;45(4):295-300.
  • 16. Güngör T, Arman Bilir Ö, Koşan Çulha V, Güngör A, Kara A, Azık FM, Yaralı HN. Retrospective evaluation of children with immune thrombocytopenic purpura and factors contributing to chronicity. Pediatr Neonatol. 2019 Aug;60(4):411-416.
  • 17. Heitink-Pollé KM, Nijsten J, Boonacker CW, de Haas M, Bruin MC. Clinical and laboratory predictors of chronic immune thrombocytopenia in children: a systematic review and meta-analysis. Blood. 2014 Nov 20;124(22):3295-307.
  • 18. Jaime-Pérez JC, Aguilar-Calderón P, Jiménez-Castillo RA, Ramos-Dávila EM, Salazar-Cavazos L, Gómez-Almaguer D. Treatment outcomes and chronicity predictors for primary immune thrombocytopenia: 10-year data from an academic center. Ann Hematol. 2020 Nov;99(11):2513-2520.
  • 19. Neunert C, Heitink-Polle KMJ, Lambert MP. A proposal for new definition (s) and management approach to paediatric refractory ITP: Reflections from the Intercontinental ITP Study Group. Br J Haematol. 2023 Oct;203(1):17-22.
  • 20. Koc BS, Ozdemir GN, Alakbarli J, Apak H, Celkan T. Experience with Pediatric Chronic Immune Thrombocytopenia over 30 Years in the Era before Eltrombopag. Children. 2024; 11(9): 1051.
  • 21. Chotsampancharoen T, Sripornsawan P, Duangchoo S, Wongchanchailert M, McNeil E. Clinical outcome of childhood chronic immune thrombocytopenia: A 38-year experience from a single tertiary center in Thailand. Pediatr Blood Cancer. 2017 Nov;64(11).
  • 22. Turhan AB, Özdemir ZC, Bör Ö. Use of Single- or Two-dose Pulse Methylprednisolone in the Treatment of Acute Immune Thrombocytopenic Purpura. Sisli Etfal Hastan Tip Bul. 2018 Mar 21;52(4):279-284.

Akut İmmun Trombositopenili Çocukların İlk Tanı Başvuru, Klinik ve Tedavi Özelliklerinin Değerlendirilmesi

Yıl 2025, Cilt: 47 Sayı: 2, 221 - 226, 27.02.2025

Öz

Primer immüntrombositopeni (ITP), daha önce idiyopatik trombositopenik purpura olarak bilinen, çocuklarda trombositopeninin en yaygın nedenidir ve diğer etiyolojiler olmaksızın izole bir düşük trombosit sayısı (<100,000/mm³) ile tanımlanır. Yıllık görülme sıklığı yaklaşık 100,000 çocukta 2 ila 5 arasıdır. Akut ITP, tanı anında mukozal kanama veya cilt bulguları ile kendini gösterir ve durumun süresine bağlı olarak akut, persistan ve kronik ITP olarak sınıflandırılır. Çocukların çoğu (%70-80), tanıdan sonra 12 ay içinde spontan iyileşme yaşar. Hem primer hem de sekonder ITP, immünolojik anormallikler sergiler, ancak sekonder vakalar belirgin etiyolojik faktörlerle ilişkilidir. Tedavi hedefleri aktif kanamayı durdurmaya ve gelecekteki kanamaları önlemeye odaklanmanın yanı sıra, sağlıkla ilgili yaşam kalitesini (HRQoL) artırmayı da kapsamaktadır. Kortikosteroidler, 1950'lerden beri ITP tedavisinin temel itici gücüdür ve son çalışmalar yüksek doz metilprednizolon (HDMP) kullanımını azaltma eğilimini göstermektedir. Bu retrospektif çalışma, Türkiye'de gerçekleştirildi ve akut ITP tanısı almış 40 pediatrik hastayı inceleyerek intravenöz metilprednizolon alan iki gruptaki değişen dozları analiz etti. Sonuçlar, her iki tedavi rejiminin benzer yanıt oranları elde ettiğini ve gruplar arasında relaps oranları veya kronik ITP gelişimi açısından anlamlı bir fark bulunmadığını gösterdi. Genel olarak, başlangıç tedavi yanıt oranlarının anlamlı şekilde farklılık göstermediği, bu çalışmanın akut ITP'nin etkili bir şekilde yönetimi için dozlama stratejilerini araştırma ihtiyacını vurguladığı belirtilmiştir. Bulgular, başlangıç tedavi aşaması için 'mega doz' metilprednizolon kullanımını desteklerken, daha düşük bir dozun da hasta sonuçlarını tehlikeye atmadan benzer şekilde etkili olabileceğini önermektedir.

Kaynakça

  • 1. Kochhar M, Neunert C. Immune thrombocytopenia: A review of upfront treatment strategies. Blood Rev. 2021 Sep;49: 100822.
  • 2. Grace RF, Lambert MP. An update on pediatric ITP: differentiating primary ITP, IPD, and PID. Blood. 2022 Aug 11;140(6): 542-555.
  • 3. Rodeghiero F, Stasi R, Gernsheimer T, Michel M, Provan D, Arnold DM, Bussel JB, Cines DB, Chong BH, Cooper N, Godeau B, Lechner K, Mazzucconi MG, McMillan R, Sanz MA, Imbach P, Blanchette V, Kühne T, Ruggeri M, George JN. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood. 2009 Mar 12;113(11): 2386-93.
  • 4. Singh G, Bansal D, Wright NAM. Immune Thrombocytopenia in Children: Consensus and Controversies. Indian J Pediatr. 2020 Feb;87(2): 150-157.
  • 5. Blanchette V, Bolton-Maggs P. Childhood immune thrombocytopenic purpura: diagnosis and management. Pediatr Clin North Am. 2008 Apr;55(2):393-420.
  • 6. Bennett CM, Neunert C, Grace RF, Buchanan G, Imbach P, Vesely SK, Kuhne T. Predictors of remission in children with newly diagnosed immune thrombocytopenia: Data from the Intercontinental Cooperative ITP Study Group Registry II participants. Pediatr Blood Cancer. 2018 Jan;65 (1).
  • 7. Sun S, Urbanus RT, Ten Cate H, de Groot PG, de Laat B, Heemskerk JWM, Roest M. Platelet Activation Mechanisms and Consequences of Immune Thrombocytopenia. Cells. 2021 Dec 1;10(12):3386.
  • 8. Moulinet T, Moussu A, Pierson L, Pagliuca S. The many facets of immune-mediated thrombocytopenia: Principles of immunobiology and immunotherapy. Blood Rev. 2023 Nov 11:101141.
  • 9. Neunert C, Terrell DR, Arnold DM, Buchanan G, Cines DB, Cooper N, Cuker A, Despotovic JM, George JN, Grace RF, Kühne T, Kuter DJ, Lim W, McCrae KR, Pruitt B, Shimanek H, Vesely SK. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019 Dec 10;3(23):3829-3866.
  • 10. Dameshek W, Rubio F Jr, Mahoney JP, Reeves WH, Burgin LA. Treatment of idiopathic thrombocytopenic purpura (ITP) with prednisone. J Am Med Assoc. 1958 Apr 12;166(15):1805-15.
  • 11. Jayabose S, Patel P, Inamdar S, Brilliant R, Mamtani R. Use of intravenous methylprednisolone in acute idiopathic thrombocytopenic purpura. Am J Pediatr Hematol Oncol. 1987 Summer;9(2): 133-5.
  • 12. Ozsoylu S, Irken G, Karabent A. High-dose intravenous methylprednisolone for acute childhood idiopathic thrombocytopenic purpura. Eur J Haematol. 1989 May;42(5): 431-5.
  • 13. Provan D, Arnold DM, Bussel JB, Chong BH, Cooper N, Gernsheimer T, Ghanima W, Godeau B, González-López TJ, Grainger J, Hou M, Kruse C, McDonald V, Michel M, Newland AC, Pavord S, Rodeghiero F, Scully M, Tomiyama Y, Wong RS, Zaja F, Kuter DJ. Updated international consensus report on the investigation and management of primary immune thrombocytopenia. Blood Adv. 2019 Nov 26;3(22):3780-3817.
  • 14. Duru F, Fisgin T, Yarali N, Kara A. Clinical course of children with immune thrombocytopenic purpura treated with intravenous immunoglobulin G or megadose methylprednisolone or observed without therapy. Pediatr Hematol Oncol. 2002 Jun;19 (4):219-25.
  • 15. Erduran E, Aslan Y, Gedik Y, Orhan F. A randomized and comparative study of intravenous immunoglobulin and mega dose methylprednisolone treatments in children with acute idiopathic thrombocytopenic purpura. Turk J Pediatr. 2003 Oct-Dec;45(4):295-300.
  • 16. Güngör T, Arman Bilir Ö, Koşan Çulha V, Güngör A, Kara A, Azık FM, Yaralı HN. Retrospective evaluation of children with immune thrombocytopenic purpura and factors contributing to chronicity. Pediatr Neonatol. 2019 Aug;60(4):411-416.
  • 17. Heitink-Pollé KM, Nijsten J, Boonacker CW, de Haas M, Bruin MC. Clinical and laboratory predictors of chronic immune thrombocytopenia in children: a systematic review and meta-analysis. Blood. 2014 Nov 20;124(22):3295-307.
  • 18. Jaime-Pérez JC, Aguilar-Calderón P, Jiménez-Castillo RA, Ramos-Dávila EM, Salazar-Cavazos L, Gómez-Almaguer D. Treatment outcomes and chronicity predictors for primary immune thrombocytopenia: 10-year data from an academic center. Ann Hematol. 2020 Nov;99(11):2513-2520.
  • 19. Neunert C, Heitink-Polle KMJ, Lambert MP. A proposal for new definition (s) and management approach to paediatric refractory ITP: Reflections from the Intercontinental ITP Study Group. Br J Haematol. 2023 Oct;203(1):17-22.
  • 20. Koc BS, Ozdemir GN, Alakbarli J, Apak H, Celkan T. Experience with Pediatric Chronic Immune Thrombocytopenia over 30 Years in the Era before Eltrombopag. Children. 2024; 11(9): 1051.
  • 21. Chotsampancharoen T, Sripornsawan P, Duangchoo S, Wongchanchailert M, McNeil E. Clinical outcome of childhood chronic immune thrombocytopenia: A 38-year experience from a single tertiary center in Thailand. Pediatr Blood Cancer. 2017 Nov;64(11).
  • 22. Turhan AB, Özdemir ZC, Bör Ö. Use of Single- or Two-dose Pulse Methylprednisolone in the Treatment of Acute Immune Thrombocytopenic Purpura. Sisli Etfal Hastan Tip Bul. 2018 Mar 21;52(4):279-284.
Toplam 22 adet kaynakça vardır.

Ayrıntılar

Birincil Dil İngilizce
Konular Çocuk Hematolojisi ve Onkolojisi
Bölüm ORİJİNAL MAKALELER / ORIGINAL ARTICLES
Yazarlar

Ersin Töret 0000-0002-6379-8326

Ezgi Baransel 0000-0002-3821-8318

Zeynep Canan Özdemir 0000-0002-9172-9627

Ozcan Bor 0000-0002-1662-3259

Yayımlanma Tarihi 27 Şubat 2025
Gönderilme Tarihi 6 Aralık 2024
Kabul Tarihi 3 Şubat 2025
Yayımlandığı Sayı Yıl 2025 Cilt: 47 Sayı: 2

Kaynak Göster

Vancouver Töret E, Baransel E, Özdemir ZC, Bor O. Evaluation of Initial Diagnosis, Clinical-Treatment Features of Children with Acute Immune Thrombocytopenia. Osmangazi Tıp Dergisi. 2025;47(2):221-6.


13299        13308       13306       13305    13307  1330126978