Research Article
BibTex RIS Cite

Efficacy of mycophenolate mofetil treatment in patients with immune thrombocytopenia who have received at least one series of treatment: a single-center study

Year 2026, Volume: 53 Issue: 1 , 103 - 107 , 10.03.2026
https://doi.org/10.5798/dicletip.1906458
https://izlik.org/JA34KD56HD

Abstract

Background: The objective of this study was to evaluate the efficacy of mycophenolate mofetil (MMF) as a second- or third-line treatment for chronic immune thrombocytopenia (ITP).
Methods: A cohort of 13 patients with chronic ITP, who had previously been treated with eltrombopag, corticosteroids, or both, without achieving an effective response, was administered mycophenolate mofetil at a daily dosage of 1000 mg. Platelet counts were monitored at the fourth and twelfth weeks of treatment.
Results: In our study, 13 patients were included, consisting of 4 females (30.8%) and 9 males (69.2%). At the 12-week mark following the initiation of MMF therapy, three out of 13 patients achieved a complete response, three out of 13 patients achieved a partial response, and seven patients showed no response. (Fig.1). At the time of transitioning to Mycophenolate Mofetil (MMF), all patients had a platelet count below 30 × 10^9/L. Approximately 40% of patients experienced an increase in platelet counts to levels above 30 × 10^9/L.
Conclusions: Mycophenolate mofetil may be considered a potential treatment option for patients with chronic ITP who are refractory to first- and second-line therapies.

Ethical Statement

The study was conducted under the decision number 301, dated 22.04.2021, from the Dicle University Faculty of Medicine Non-Interventional Clinical Research Ethics Committee.

References

  • 1.Stasi R. How to approach thrombocytopenia.Hematology. 2012(1); 191–7.
  • 2.Terrell DR, Neunert CE, Cooper N, et al. ImmuneThrombocytopenia (ITP): Current Limitations inPatient Management. Medicina. 2020; 56(12):667.
  • 3. González-López T. J, Newland A, Provan D. CurrentConcepts in the Diagnosis and Management of AdultPrimary Immune Thrombocytopenia: Our PersonalView. Medicina. 2023; 59(4): 815.
  • 4.Liu X, Hou Y, Hou M. How we treat primaryimmune thrombocytopenia in adults. J. Hematol.Oncol.J Hematol Oncol. 2023; 16 (4): 1-20.
  • 5. Martínez-Carballeira D, Bernardo Á, Caro A, Soto I, Gutiérrez L. Pathophysiology, Clinical Manifestations and Diagnosis of Immune Thrombocytopenia: Contextualization from a Historical Perspective. Hematol Rep. 2024 Apr 3;16(2):204-19.
  • 6.Al-Samkari H, Kuter, DJ. Immunethrombocytopenia in adults: modern approaches todiagnosis and treatment, Semin. Thromb. Hemost.2020;46(3): 275–88.
  • 7.Cohen YC, Djulbegovic B, Shamai-Lubovitz O,Mozes B. The Bleeding Risk and Natural History ofIdiopathic Thrombocytopenic Purpura in Patientswith Persistent Low Platelet Counts. Arch. Intern.Med. 2000;160(3): 1630–8.
  • 8.Allison AC, Eugui EM. Purine metabolism andimmunosuppressive effects of mycophenolatemofetil (MMF). Clin. Transplant. 1996; 10: 77–84.
  • 9.Hou M, Peng J, Shi Y, et al. Mycophenolate mofetil(MMF) for the treatment of steroid-resistantidiopathic thrombocytopenic purpura. Eur JHaematol. 2003;70(6):353-7.
  • 10.K. Kojouri, S.K. Vesely, D.R. Terrell, J.N. GeorgeSplenectomy for adult patients with idiopathicthrombocytopenic purpura: a systematic review toassess long-term platelet count responses,prediction of response, and surgical complicationsBlood, 104 (2004), pp. 2623-34.
  • 11.Audia S., Mahevas M., Samson M., Godeau B.,Bonnotte B. Pathogenesis of immunethrombocytopenia. Autoimmun. Rev. 2017; 16:620–32.
  • 12.Bradbury CA, Pell J, Hill Q, et al. Mycophenolatemofetil for first-line treatment of immunethrombocytopenia. New England J Med.2021;385(10):885–95.
  • 13.Abdelwahab OA, Mechi A, Gahlan S, et al. Efficacyand safety of mycophenolate mofetil in patients withimmune thrombocytopenic purpura: a systematicreview and meta-analysis. Clin. Rheumatol. 2024;43(2): 621–32.
  • 14.Zhang W. Ji L, Cao X, Chen Y, et al. Mycophenolatemofetil as a treatment for refractory idiopathicthrombocytopenic purpura. Acta Pharmacol. Sin.2005; 26(5): 598–602.
  • 15. Taylor A, Neave L, Solanki S, et al. Mycophenolate mofetil therapy for severe immune thrombocytopenia. Br J Haematol. 2015;171(4):625-30.

En az bir seri tedavi almış immun trombositopeni tanılı hastalarda mikofenolat mofetil tedavisinin etkinliği: tek merkez çalışması

Year 2026, Volume: 53 Issue: 1 , 103 - 107 , 10.03.2026
https://doi.org/10.5798/dicletip.1906458
https://izlik.org/JA34KD56HD

Abstract

Amaç: Bu çalışmanın amacı, kronik immün trombositopeni (İTP) tedavisinde ikinci veya üçüncü basamak tedavi olarak mikofenolat mofetilin (MMF) etkinliğini değerlendirmektir.
Yöntemler: Daha önce eltrombopag, kortikosteroidler veya her ikisiyle tedavi edilmiş ve etkili bir yanıt alınamamış kronik İTP'li 13 hastadan oluşan bir kohorta, günlük 1000 mg mikofenolat mofetil uygulandı. Trombosit sayıları, tedavinin dördüncü ve on ikinci haftalarında izlendi.
Bulgular: Çalışmamıza 4 kadın (%30,8) ve 9 erkek (%69,2) olmak üzere toplam 13 hasta dahil edildi. MMF tedavisinin başlamasından sonraki 12. haftada, 13 hastanın 3'ünde tam yanıt, 13 hastanın 3'ünde yanıt ve 7 hastada yanıt alınamamıştır. Mikofenolat Mofetil'e (MMF) geçiş sırasında tüm hastaların trombosit sayısı 30 × 10^9/L'nin altındaydı. Hastaların yaklaşık %40'ında trombosit sayılarında 30 × 10^9/L'nin üzerine çıkan bir artış görüldü.
Sonuç: Mikofenolat mofetil, birinci ve ikinci basamak tedavilere dirençli kronik İTP'li hastalar için potansiyel bir tedavi seçeneği olarak düşünülebilir.

References

  • 1.Stasi R. How to approach thrombocytopenia.Hematology. 2012(1); 191–7.
  • 2.Terrell DR, Neunert CE, Cooper N, et al. ImmuneThrombocytopenia (ITP): Current Limitations inPatient Management. Medicina. 2020; 56(12):667.
  • 3. González-López T. J, Newland A, Provan D. CurrentConcepts in the Diagnosis and Management of AdultPrimary Immune Thrombocytopenia: Our PersonalView. Medicina. 2023; 59(4): 815.
  • 4.Liu X, Hou Y, Hou M. How we treat primaryimmune thrombocytopenia in adults. J. Hematol.Oncol.J Hematol Oncol. 2023; 16 (4): 1-20.
  • 5. Martínez-Carballeira D, Bernardo Á, Caro A, Soto I, Gutiérrez L. Pathophysiology, Clinical Manifestations and Diagnosis of Immune Thrombocytopenia: Contextualization from a Historical Perspective. Hematol Rep. 2024 Apr 3;16(2):204-19.
  • 6.Al-Samkari H, Kuter, DJ. Immunethrombocytopenia in adults: modern approaches todiagnosis and treatment, Semin. Thromb. Hemost.2020;46(3): 275–88.
  • 7.Cohen YC, Djulbegovic B, Shamai-Lubovitz O,Mozes B. The Bleeding Risk and Natural History ofIdiopathic Thrombocytopenic Purpura in Patientswith Persistent Low Platelet Counts. Arch. Intern.Med. 2000;160(3): 1630–8.
  • 8.Allison AC, Eugui EM. Purine metabolism andimmunosuppressive effects of mycophenolatemofetil (MMF). Clin. Transplant. 1996; 10: 77–84.
  • 9.Hou M, Peng J, Shi Y, et al. Mycophenolate mofetil(MMF) for the treatment of steroid-resistantidiopathic thrombocytopenic purpura. Eur JHaematol. 2003;70(6):353-7.
  • 10.K. Kojouri, S.K. Vesely, D.R. Terrell, J.N. GeorgeSplenectomy for adult patients with idiopathicthrombocytopenic purpura: a systematic review toassess long-term platelet count responses,prediction of response, and surgical complicationsBlood, 104 (2004), pp. 2623-34.
  • 11.Audia S., Mahevas M., Samson M., Godeau B.,Bonnotte B. Pathogenesis of immunethrombocytopenia. Autoimmun. Rev. 2017; 16:620–32.
  • 12.Bradbury CA, Pell J, Hill Q, et al. Mycophenolatemofetil for first-line treatment of immunethrombocytopenia. New England J Med.2021;385(10):885–95.
  • 13.Abdelwahab OA, Mechi A, Gahlan S, et al. Efficacyand safety of mycophenolate mofetil in patients withimmune thrombocytopenic purpura: a systematicreview and meta-analysis. Clin. Rheumatol. 2024;43(2): 621–32.
  • 14.Zhang W. Ji L, Cao X, Chen Y, et al. Mycophenolatemofetil as a treatment for refractory idiopathicthrombocytopenic purpura. Acta Pharmacol. Sin.2005; 26(5): 598–602.
  • 15. Taylor A, Neave L, Solanki S, et al. Mycophenolate mofetil therapy for severe immune thrombocytopenia. Br J Haematol. 2015;171(4):625-30.
There are 15 citations in total.

Details

Primary Language English
Subjects Health Care Administration, Medical Education, Health Services and Systems (Other)
Journal Section Research Article
Authors

Abdullah Karakuş

Vehbi Demircan

Orhan Ayyıldız

Submission Date December 15, 2025
Acceptance Date February 13, 2026
Publication Date March 10, 2026
DOI https://doi.org/10.5798/dicletip.1906458
IZ https://izlik.org/JA34KD56HD
Published in Issue Year 2026 Volume: 53 Issue: 1

Cite

APA Karakuş, A., Demircan, V., & Ayyıldız, O. (2026). Efficacy of mycophenolate mofetil treatment in patients with immune thrombocytopenia who have received at least one series of treatment: a single-center study. Dicle Medical Journal, 53(1), 103-107. https://doi.org/10.5798/dicletip.1906458
AMA 1.Karakuş A, Demircan V, Ayyıldız O. Efficacy of mycophenolate mofetil treatment in patients with immune thrombocytopenia who have received at least one series of treatment: a single-center study. Dicle Medical Journal. 2026;53(1):103-107. doi:10.5798/dicletip.1906458
Chicago Karakuş, Abdullah, Vehbi Demircan, and Orhan Ayyıldız. 2026. “Efficacy of Mycophenolate Mofetil Treatment in Patients With Immune Thrombocytopenia Who Have Received at Least One Series of Treatment: A Single-Center Study”. Dicle Medical Journal 53 (1): 103-7. https://doi.org/10.5798/dicletip.1906458.
EndNote Karakuş A, Demircan V, Ayyıldız O (March 1, 2026) Efficacy of mycophenolate mofetil treatment in patients with immune thrombocytopenia who have received at least one series of treatment: a single-center study. Dicle Medical Journal 53 1 103–107.
IEEE [1]A. Karakuş, V. Demircan, and O. Ayyıldız, “Efficacy of mycophenolate mofetil treatment in patients with immune thrombocytopenia who have received at least one series of treatment: a single-center study”, Dicle Medical Journal, vol. 53, no. 1, pp. 103–107, Mar. 2026, doi: 10.5798/dicletip.1906458.
ISNAD Karakuş, Abdullah - Demircan, Vehbi - Ayyıldız, Orhan. “Efficacy of Mycophenolate Mofetil Treatment in Patients With Immune Thrombocytopenia Who Have Received at Least One Series of Treatment: A Single-Center Study”. Dicle Medical Journal 53/1 (March 1, 2026): 103-107. https://doi.org/10.5798/dicletip.1906458.
JAMA 1.Karakuş A, Demircan V, Ayyıldız O. Efficacy of mycophenolate mofetil treatment in patients with immune thrombocytopenia who have received at least one series of treatment: a single-center study. Dicle Medical Journal. 2026;53:103–107.
MLA Karakuş, Abdullah, et al. “Efficacy of Mycophenolate Mofetil Treatment in Patients With Immune Thrombocytopenia Who Have Received at Least One Series of Treatment: A Single-Center Study”. Dicle Medical Journal, vol. 53, no. 1, Mar. 2026, pp. 103-7, doi:10.5798/dicletip.1906458.
Vancouver 1.Abdullah Karakuş, Vehbi Demircan, Orhan Ayyıldız. Efficacy of mycophenolate mofetil treatment in patients with immune thrombocytopenia who have received at least one series of treatment: a single-center study. Dicle Medical Journal. 2026 Mar. 1;53(1):103-7. doi:10.5798/dicletip.1906458