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Allogeneic stem cell transplantation in patients with myelofibrosis: single center, retrospective study

Year 2020, , 109 - 112, 30.06.2020
https://doi.org/10.19161/etd.756246

Abstract

Aim: Primary myelofibrosis (PMF) is a clonal stem cell disease characterized by chronic myeloproliferation, atypical megakaryocytic hyperplasia and bone marrow fibrosis. Allogeneic stem cell transplantation, which is the only curative treatment option, is recommended for patients diagnosed with DIPSS moderate-2 or high-risk primary or secondary myelofibrosis according to the Turkish Ministry of Health indication list. In this study, we aimed to share the follow-up data after allogeneic transplantation of our patients with myelofibrosis.
Materials and Methods: This retrospective and cross-sectional evaluation included 9 cases of myelofibrosis that underwent allogeneic transplantation between 2011 and 2016 at Şişli Florence Nightingale Hospital Hematopoietic Stem Cell Transplantation Center. All demographic characteristics and parameters such as DIPPS-plus scores, engraftment times, frequency of graft versus host disease, and survival data were recorded.
Results: The mean age of the patients was 49.7 (34-63) years. The mean time from diagnosis to transplantation was 30.2 months (1.7-65.2). Matched sibling donor was used in 6 cases and matched unrelated donor in 3 cases. It was observed that neutrophil engraftment time was significantly shorter than matched unrelated donor transplantation when compare to matched related donor transplantation, whereas platelet engraftment times did not show a statistically significance. Acute GVHD developed in 3 patients (33.4%) and chronic GVHD in 6 patients (66.6%). Cumulative survival is 70% for 5 years.
Discussion: In our center, the overall survival rate in patients with myelofibrosis after allogeneic transplantation seems to be high compared to many series in the literature. Although the small number of patients is an important limitation, there is both age and donor variety. On the other hand, the fact that GVHD was seen at a higher frequency compared to the literature and the survival rate remains high, which emphasizes the importance of post-transplant patient follow-up and good management of the complications.

References

  • Tefferi A. Primary myelofibrosis: 2019 update on diagnosis, risk-stratification and management. Am J Hematol. 2018; 93: 1551-60.
  • Tefferi A, Guglielmelli P, Larson DR, Finke C, Wassie EA, Pieri L, et al. Long-term survival and blast transformation in molecularly annotated essential thrombocythemia, polycythemia vera, and myelofibrosis. Blood. 2014; 124: 2507-13; quiz 615.
  • Cervantes F, Dupriez B, Pereira A, Passamonti F, Reilly JT, Morra E, et al. New prognostic scoring system for primary myelofibrosis based on a study of the International Working Group for Myelofibrosis Research and Treatment. Blood. 2009; 113: 2895-901.
  • Passamonti F, Cervantes F, Vannucchi AM, Morra E, Rumi E, Cazzola M, et al. Dynamic International Prognostic Scoring System (DIPSS) predicts progression to acute myeloid leukemia in primary myelofibrosis. Blood. 2010; 116: 2857-8.
  • Gangat N, Caramazza D, Vaidya R, George G, Begna K, Schwager S, et al. DIPSS plus: a refined Dynamic International Prognostic Scoring System for primary myelofibrosis that incorporates prognostic information from karyotype, platelet count, and transfusion status. J Clin Oncol. 2011; 29: 392-7.
  • Lavi N, Rowe JM, Zuckerman T. Allogeneic stem-cell transplantation for myelofibrosis. Curr Opin Hematol. 2017; 24: 475-80.
  • Kroger N, Giorgino T, Scott BL, Ditschkowski M, Alchalby H, Cervantes F, et al. Impact of allogeneic stem cell transplantation on survival of patients less than 65 years of age with primary myelofibrosis. Blood. 2015; 125: 3347-50; quiz 64.
  • Kroger NM, Deeg JH, Olavarria E, Niederwieser D, Bacigalupo A, Barbui T, et al. Indication and management of allogeneic stem cell transplantation in primary myelofibrosis: a consensus process by an EBMT/ELN international working group. Leukemia. 2015; 29: 2126-33.
  • Kroger N, Holler E, Kobbe G, Bornhauser M, Schwerdtfeger R, Baurmann H, et al. Allogeneic stem cell transplantation after reduced-intensity conditioning in patients with myelofibrosis: a prospective, multicenter study of the Chronic Leukemia Working Party of the European Group for Blood and Marrow Transplantation. Blood. 2009; 114: 5264-70.
  • Devlin R, Gupta V. Myelofibrosis: to transplant or not to transplant? Hematology Am Soc Hematol Educ Program. 2016; 2016: 543-51.
  • Shanavas M, Popat U, Michaelis LC, Fauble V, McLornan D, Klisovic R, et al. Outcomes of Allogeneic Hematopoietic Cell Transplantation in Patients with Myelofibrosis with Prior Exposure to Janus Kinase 1/2 Inhibitors. Biol Blood Marrow Transplant. 2016; 22: 432-40.
  • Schmohl JU, Groh C, Faul C, Vogel W, Mohle R, Wirths S, et al. Allogeneic hematopoietic cell transplantation in patients with myelofibrosis: A single center experience. Ann Hematol. 2016; 95: 973-83.
  • Robin M, de Wreede LC, Wolschke C, Schetelig J, Eikema DJ, Van Lint MT, et al. Long-term outcome after allogeneic hematopoietic cell transplantation for myelofibrosis. Haematologica 2019 Sep;104(9):1782-8. doi: 10.3324/haematol.2018.205211

Miyelofibrozisli olgularda allojenik kök hücre nakil sonuçlarımız: tek merkezli, retrospektif bir analiz

Year 2020, , 109 - 112, 30.06.2020
https://doi.org/10.19161/etd.756246

Abstract

Amaç: Primer miyelofibrozis (PMF), kronik miyeloproliferasyon, atipik megakaryositik hiperplazi ve kem k l ğ f broz s le karakter ze klonal b r kök hücre hastalığıdır. Bu hastalarda günümüzde halen göster leb lm ş tek şifa sağlayıcı tedavi seçeneği olan allojenik kök hücre nakli, ülkemiz Sağlık Bakanlığı endikasyon listesine göre DIPPS skoru orta-2 veya yüksek riskli birincil veya ikincil miyelofibrozisli hastalara önerilmektedir. Çalışmamızda miyelofibrozisli hastalarımızın allojenik nakil sonrası takip verilerini paylaşmayı amaçladık.
Gereç ve Yöntem: Geriye dönük ve kesitsel bu değerlendirmeye Şişli Florence Nightingale Hastanesi Hematopoetik Kök Hücre Nakil Merkezinde 2011-2016 yılları arasında allojenik nakil yapılan ve takipte olan toplam dokuz miyelofibrozis tanılı olgu dahil edildi. Olguların tüm demografik özellikleri yanı sıra, DIPPS-plus skorları, engrafman süreleri, graft versus host hastalığı (GVHH) görülme sıklıkları gibi parametreler ve sağ kalım verileri kaydedildi.
Bulgular: Olguların ortalama yaşları 49,7 (34-63) yıl idi. Tanı ile transplant arası geçen süre ortalama 30,2 ay (1,7-65,2) idi. Tam uyumlu kardeş nakil altı olguda ve akraba dışı nakil üç olguda yapıldı. Tam uyumlu kardeş nakilde, nötrofil engraftman süresinin akraba dışı nakile göre anlamlı kısa iken, trombosit engraftman sürelerinin ise tam uyumlu kardeş nakilde kısa olma eğilimi ile birlikte istatistiksel anlamlı farklılık göstermediği izlenmiştir. Toplam dokuz hastanın takibinde üç olguda (%33,4) akut, altı olguda (%66,6) kronik GVHH geliştiği izlenmiştir. Kümülatif sağ kalım beş yıllık %70’dir.
Sonuç: Merkezimizde miyelofibrozisli olguların değerlendirmelerinde, özellikle toplam sağ kalım oranı literatürdeki birçok seriye göre yüksek görünmektedir. Hasta sayısının az oluşu önemli bir sınırlama olmakla birlikte hem yaş hem de verici çeşitliliği mevcuttur. Ayrıca yüksek GVHH oranlarına karşın sağ kalımın yüksek kalmasının, hasta takibinin ve komplikasyonların iyi yönetiminin önemini vurguladığını düşünüyoruz.

References

  • Tefferi A. Primary myelofibrosis: 2019 update on diagnosis, risk-stratification and management. Am J Hematol. 2018; 93: 1551-60.
  • Tefferi A, Guglielmelli P, Larson DR, Finke C, Wassie EA, Pieri L, et al. Long-term survival and blast transformation in molecularly annotated essential thrombocythemia, polycythemia vera, and myelofibrosis. Blood. 2014; 124: 2507-13; quiz 615.
  • Cervantes F, Dupriez B, Pereira A, Passamonti F, Reilly JT, Morra E, et al. New prognostic scoring system for primary myelofibrosis based on a study of the International Working Group for Myelofibrosis Research and Treatment. Blood. 2009; 113: 2895-901.
  • Passamonti F, Cervantes F, Vannucchi AM, Morra E, Rumi E, Cazzola M, et al. Dynamic International Prognostic Scoring System (DIPSS) predicts progression to acute myeloid leukemia in primary myelofibrosis. Blood. 2010; 116: 2857-8.
  • Gangat N, Caramazza D, Vaidya R, George G, Begna K, Schwager S, et al. DIPSS plus: a refined Dynamic International Prognostic Scoring System for primary myelofibrosis that incorporates prognostic information from karyotype, platelet count, and transfusion status. J Clin Oncol. 2011; 29: 392-7.
  • Lavi N, Rowe JM, Zuckerman T. Allogeneic stem-cell transplantation for myelofibrosis. Curr Opin Hematol. 2017; 24: 475-80.
  • Kroger N, Giorgino T, Scott BL, Ditschkowski M, Alchalby H, Cervantes F, et al. Impact of allogeneic stem cell transplantation on survival of patients less than 65 years of age with primary myelofibrosis. Blood. 2015; 125: 3347-50; quiz 64.
  • Kroger NM, Deeg JH, Olavarria E, Niederwieser D, Bacigalupo A, Barbui T, et al. Indication and management of allogeneic stem cell transplantation in primary myelofibrosis: a consensus process by an EBMT/ELN international working group. Leukemia. 2015; 29: 2126-33.
  • Kroger N, Holler E, Kobbe G, Bornhauser M, Schwerdtfeger R, Baurmann H, et al. Allogeneic stem cell transplantation after reduced-intensity conditioning in patients with myelofibrosis: a prospective, multicenter study of the Chronic Leukemia Working Party of the European Group for Blood and Marrow Transplantation. Blood. 2009; 114: 5264-70.
  • Devlin R, Gupta V. Myelofibrosis: to transplant or not to transplant? Hematology Am Soc Hematol Educ Program. 2016; 2016: 543-51.
  • Shanavas M, Popat U, Michaelis LC, Fauble V, McLornan D, Klisovic R, et al. Outcomes of Allogeneic Hematopoietic Cell Transplantation in Patients with Myelofibrosis with Prior Exposure to Janus Kinase 1/2 Inhibitors. Biol Blood Marrow Transplant. 2016; 22: 432-40.
  • Schmohl JU, Groh C, Faul C, Vogel W, Mohle R, Wirths S, et al. Allogeneic hematopoietic cell transplantation in patients with myelofibrosis: A single center experience. Ann Hematol. 2016; 95: 973-83.
  • Robin M, de Wreede LC, Wolschke C, Schetelig J, Eikema DJ, Van Lint MT, et al. Long-term outcome after allogeneic hematopoietic cell transplantation for myelofibrosis. Haematologica 2019 Sep;104(9):1782-8. doi: 10.3324/haematol.2018.205211
There are 13 citations in total.

Details

Primary Language Turkish
Subjects Health Care Administration
Journal Section Research Articles
Authors

Deniz Gören Şahin 0000-0002-3189-6810

Nurcan Özçelik 0000-0002-3076-3223

Burcu Kurt Yıldırım 0000-0002-2617-1620

Fehmi Hindilerden

Serkan Güvenç 0000-0002-4476-2651

Hasan Sami Göksoy 0000-0003-3795-3502

Reyhan Diz Küçükkaya 0000-0001-5814-7118

Emine Tülay Özçelik 0000-0002-2859-6032

Mutlu Arat 0000-0003-2039-8557

Publication Date June 30, 2020
Submission Date July 17, 2019
Published in Issue Year 2020

Cite

Vancouver Şahin DG, Özçelik N, Kurt Yıldırım B, Hindilerden F, Güvenç S, Göksoy HS, Diz Küçükkaya R, Özçelik ET, Arat M. Miyelofibrozisli olgularda allojenik kök hücre nakil sonuçlarımız: tek merkezli, retrospektif bir analiz. ETD. 2020;59(2):109-12.

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