Evaluation of Pediatric Patients with Immune Thrombocytopenia Regarding Clinical Course and Treatment Response: A Retrospective Single-Center Experience

Abstract

Aim: Immune thrombocytopenia (ITP) is the most common cause of acquired thrombocytopenia in childhood. It is an autoimmune disease that is elicited by the destruction of platelets with autoantibodies. We aimed in this study to investigate the clinical features of the disease and provide insight into how the treatment method is determined accordingly. It is also intended to examine and compare the treatment responses. Materials and Methods: Between January 2013 and December 2018, a total of 80 patients diagnosed as ITP, at the Pediatric Hematology Outpatient Clinic were enrolled in the study. During the study period, we provided to record data of all patients thoroughly. We finally compared the results of those undergoing appropriate medical therapy statistically after the exclusion of the causes of secondary thrombocytopenia. Results: The mean age of the patients was 5 ± 2.3 years; the ratio of females to males was found to be 1 to 2. Among the most common complaints were that ecchymosis (63.7%) ranked first, followed by petechiae (%20). Four (5%) patients received no treatment. Methylprednisolone, IVIG, and dexamethasone were given in 49 (61.2%), 17 (21.2%), and 8 (10%) patients, respectively. Both of methylprednisolone and IVIG were given to only 2 (2.5%) patients. Conclusion: The ITP patients receiving IVIG have been found to progress to chronicity more than those receiving methylprednisolone. Our study also presents knowledge to be able to predict the development of chronicity in ITP.

Keywords

• Immune thrombocytopenic purpura
• thrombocytopenia
• IVIG
• steroid treatment

İmmün Trombositopenili Pediatrik Hastaların Klinik Seyir ve Tedavi Yanıtı Açısından Değerlendirilmesi: Retrospektif Tek Merkez Deneyimi

Abstract

Amaç: İmmün trombositopeni (ITP), çocukluk çağında edinilmiş trombositopeninin en yaygın nedenidir. Otoantikorlar ile trombositlerin yok edilmesiyle ortaya çıkan otoimmün bir hastalıktır. Bu çalışmada, hastalığın klinik özelliklerini ve buna göre tedavi yönteminin nasıl belirlendiğini araştırmayı amaçladık. Ayrıca tedavi yanıtlarının incelenmesi ve karşılaştırılması amaçlanmaktadır. Gereç ve Yöntem: Ocak 2013- Aralık 2018 tarihleri arasında Çocuk Hematoloji Polikliniği'nde İTP tanısı almış toplam 80 hasta çalışmaya alındı. Çalışma süresi boyunca tüm hastaların verilerinin eksiksiz bir şekilde kayıt altına alınmasını sağladık. İkincil trombositopeni nedenleri dışlandıktan sonra son olarak uygun tıbbi tedavi görenlerin sonuçlarını istatistiksel olarak karşılaştırdık. Bulgular: Hastaların ortalama yaşı 5 ± 2.3 yıldı; Kadınların erkeklere oranı 1’e 2 olarak bulundu. En sık görülen şikayetler arasında ilk sırada ekimoz (%63,7), ardından peteşi (%20) vardı. Dört (% 5) hasta tedavi almadı. Metilprednizolon, İVİG ve deksametazon sırasıyla 49 (% 61,2), 17 (% 21,2) ve 8 (% 10) hastaya verildi. Hem metilprednizolon hem de IVIG sadece 2 (% 2,5) hastaya verildi. Sonuç: IVIG alan ITP hastalarının, metilprednizolon alanlara göre kronikleşmeye daha fazla ilerlediği bulunmuştur. Çalışmamız ayrıca ITP'deki kronikliğin gelişimini tahmin etmek için de bilgi sunmaktadır.

Keywords

• İmmün trombositopenik purpura
• trombositopeni
• IVIG
• steroid tedavisi

References

1. 1. Rodeghiero F, Stasi R, Gernsheimer T. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood, 2009; 113:2386- 2393.
2. 2. Pels SG. Current therapies in primary immune thrombocytopenia. Semin Thromb Hemost, 2011; 37:621-30.
3. 3. Platelet Disorders of Infancy and Childhood. Nathan and Oski (eds) Haematology of Infancy and Childhood, 1993:1552-1580.
4. 4. Acquired Platelet Defects. Nathan and Oski (eds) Haematology of Infancy and Childhood, Sixth Edition. Saunders Company, Philadelphia, 2003:1597-1609.
5. 5. George JN, Woolf SH, Raskob GE. Idiopathic thrombocytopenic purpura: a practice guideline developed by explicit methods for the American Society of Hematology. Blood, 1996; 88:3-40.
6. 6. Neunert C, Lim W, Crowther M. The American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia. Blood, 2011; 117:4190-4207.
7. 7. Newland A, Lee EJ, McDonald V, Bussel JB. Fostamatinib for persistent/chronic adult immune thrombocytopenia. Immunotherapy, .2018; 10(1):9-25.
8. 8. Güngör T, Bilir ÖA, Çulha VK, et al. Retrospective evaluation of children with immune thrombocytopenic purpura and factors contributing to chronicity. Pediatrics & Neonatology., 2018.

9. 9. Neunert CE, Buchanan GR, Imbach P, et al. Intercontinental Childhood ITP Study Group Registry II Participants. Severe hemorrhage in children with newly diagnosed immune thrombocytopenic purpura. Blood, 2008; 112(10):4003-4008.
10. 10. Imbach P, Kühne T, Müller D, et al. Childhood ITP: 12 months follow‐up data from the prospective registry I of the Intercontinental Childhood ITP Study Group (ICIS). Pediatric blood & cancer, ) 2006; 46(3):351-356.
11. 11. Altıntop L, Albayrak D. Oral high‐dose methylprednisolone and intravenous immunoglobulin treatments in adult chronic idiopathic thrombocytopenic purpura. American journal of hematology, 1997; 56(3):191-192.