Deniz Gören ŞAHİN, Nurcan ÖZÇELİK, Burcu Kurt YILDIRIM, Fehmi HİNDİLERDEN, Serkan GÜVENÇ, Hasan Sami GÖKSOY, Diz Reyhan KÜÇÜKKAYA, Emine Tülay ÖZÇELİK, Mutlu ARAT

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

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.

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

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.

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