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

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 Sisli 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.

Amaç: Primer miyelofibrozis (PMF), kronik miyeloproliferasyon, atipik megakaryositik hiperplazi ve kem k l g f broz s le karakter ze klonal b r kök hücre hastaligidir. Bu hastalarda günümüzde halen göster leb lm s tek sifa saglayici tedavi seçenegi olan allojenik kök hücre nakli, ülkemiz Saglik Bakanligi endikasyon listesine göre DIPPS skoru orta-2 veya yüksek riskli birincil veya ikincil miyelofibrozisli hastalara önerilmektedir. Çalismamizda miyelofibrozisli hastalarimizin allojenik nakil sonrasi takip verilerini paylasmayi amaçladik. Gereç ve Yöntem: Geriye dönük ve kesitsel bu degerlendirmeye Sisli Florence Nightingale Hastanesi Hematopoetik Kök Hücre Nakil Merkezinde 2011-2016 yillari arasinda allojenik nakil yapilan ve takipte olan toplam dokuz miyelofibrozis tanili olgu dahil edildi. Olgularin tüm demografik özellikleri yani sira, DIPPS-plus skorlari, engrafman süreleri, graft versus host hastaligi (GVHH) görülme sikliklari gibi parametreler ve sag kalim verileri kaydedildi. Bulgular: Olgularin ortalama yaslari 49,7 (34-63) yil idi. Tani ile transplant arasi geçen süre ortalama 30,2 ay (1,7-65,2) idi. Tam uyumlu kardes nakil alti olguda ve akraba disi nakil üç olguda yapildi. Tam uyumlu kardes nakilde, nötrofil engraftman süresinin akraba disi nakile göre anlamli kisa iken, trombosit engraftman sürelerinin ise tam uyumlu kardes nakilde kisa olma egilimi ile birlikte istatistiksel anlamli farklilik göstermedigi izlenmistir. Toplam dokuz hastanin takibinde üç olguda (%33,4) akut, alti olguda (%66,6) kronik GVHH gelistigi izlenmistir. Kümülatif sag kalim bes yillik %70’dir. Sonuç: Merkezimizde miyelofibrozisli olgularin degerlendirmelerinde, özellikle toplam sag kalim orani literatürdeki birçok seriye göre yüksek görünmektedir. Hasta sayisinin az olusu önemli bir sinirlama olmakla birlikte hem yas hem de verici çesitliligi mevcuttur. Ayrica yüksek GVHH oranlarina karsin sag kalimin yüksek kalmasinin, hasta takibinin ve komplikasyonlarin iyi yönetiminin önemini vurguladigini düsünüyoruz.