Ekstrakraniyal malign germ hücreli tümör tanılı hastaların klinik özellikleri ve tedavi sonuçları; 20 yıllık tek merkez deneyimi

Introduction: Germ cell tumors account for 2–3% of all pediatric tumors. The aim of this study was to evaluate the clinical features and treatment outcomes of pediatric patients treated and followed up for extragonadal MGCTs in our center. Materials and Methods: A total of 41 patients diagnosed with MGCTs in the pediatric oncology department of Akdeniz University between June 1999 and June 2019 were evaluated retrospectively. Results: Twenty-nine (71%) of the patients were girls and female dominance (p<0.001). The median age was 3.22 (0–18) years. The most patients in the ≤ 5year age group (p<0.001). Nineteen (44%) of the tumors were gonadal and 22 (54%) were extragonadal. The most common histolology of MGCTs were yolk sac tumor (36%), mixed GCTs (29%), immature teratoma (20%), and dysgerminoma (15%). Twenty-five (61%) patients presented with advanced stage disease and 37 patients (90%) were treated with chemotherapy. The patients with stage I testicular and stage I ovarian germ cell tumors underwent complete tumor resection followed by a watch-and-wait approach with alpha fetoprotein monitoring without chemotherapy. Of six patients with relapse/refractory disease, two patients survived. Two patients who underwent autologous stem cell transplantation showed complete response but later died due to infection. The median follow-up period of the patients was 34.9 (4–190.6) months and the 10-year overall and disease-free survival rates were 77.1±6.8% 77.1±6.8%. Two relapsed refractory patients who underwent autologous transplantation survived at a mean of 33.21 months. Conclusions: The clinical features and treatment outcomes of the patients in our study were consistent with the literature. The fact that most of our patients were symptomatic at presentation and had advanced stage disease when diagnosed highlights the importance of detailed evaluation and examination. Although good outcomes are achieved in patients with early stage disease, new treatment approaches are needed for patients with advanced and relapsing diseaseGiriş: Germ hücreli tümör tüm pediatrik tümörlerin %2-3’ünü oluşturur. Özellikle platin bazlı kemoterapi rejimlerinin uygulanmasından sonra sağ kalım oranları %85’lerden fazladır. Malign germ hücreli tümörler (MGHT) çocuklarda oldukça heterojen bir gruptur. Bu çalışma ile ekstrakraniyal MGHT tanılı hastalarımızın klinik özellikleri ve tedavi sonuçlarının değerlendirilmesi amaçlandı. Gereç ve Yöntem: Akdeniz Üniversitesi Çocuk Onkoloji Kliniği’nde 1999 –2019 Haziran tarihleri arasında ekstrakraniyal MGHT tanısı alan 41 hasta geriye dönük olarak değerlendirildi. Bulgular: Hastaların 29 (%71) `i kız olup K/E cinsiyet oranı: 1,75 olup anlamlı olarak kız cinsiyet hakimdi (p<0.001). Ortanca tanı yaşı 3,22 yıl (0-18 yaş) olup hastalar ağırlıklı olarak (%56 hasta) ≤ 5 yaş idi (p<0.001). Tümörlerin 19 (%44) `ü gonadal, 22 (%54) `ü ekstragonadal olup en sık ekstagonadal yerleşim yeri sakrokoksigeal bölge (%22) idi. Histolojik değerlendirmede sırasıyla yolk sak tümörü (%36), mikst GHT (%29), immatür teratom (%20) ve disgerminom (%15) saptandı. Hastaların 25 (%61)`i ileri evre hastalık ile başvurmuştu. Hastaların 37 (%90)’ına kemoterapi verildi. Evre I testis ve evre I over GHT hastalarında tümörün cerrahi olarak tam çıkartılmasının ardından αFP değerleri takip edilerek “bekle ve izle” yaklaşımı ile kemoterapi verilmedi. Tanı sonrası relaps refrakter hastalık ile seyreden 6 hastanın ikisi progresif hastalıktan kaybedildi. Otolog kök hücre nakli yapılan iki hastada nakil sonrası kür sağlanmasına rağmen enfeksiyon nedeni ile kaybedildi. Hastaların ortanca izlem süresi 34.9 ay (4-190,6 ay), 5 ve 10 yıllık genel ve hastalıksız yaşam oranları 81.9±6.3%, 81.9±6.3% ve 77.1±6.8% 77.1±6.8 ve %77,1±6,8 olarak bulundu. Nakil yapılan iki hastanın sağkalım süresi ortalama 33.21 ay olarak hesaplandı. Sonuç: Ekstrakraniyal MGHT`lerin tedavisinde, konservatif cerrahi, evre I hastalar için “bekle ve gör” yaklaşımı ve platin bazlı kemoterapi rejimleri ile başarılı sonuçlar alınmaktadır. İlk başvuruda hastaların yakınmalarının olmasına rağmen çoğu hastanın ileri evre hastalık ile başvurduğunun saptanması hekimlerin ayrıntılı değerlendirme ve muayenelerinin önemine dikkat çekmektedir. Erken evre hastalarda sonuçlar başarılı iken ileri evre ve relaps hastalarda yeni tedavi yaklaşımlarına ihtiyaç vardır

Paroxysmal nocturnal hemoglobinuria case with thrombosis under eculizumab treatment

43. Ulusal Hematoloji Kongresi’nde sunulmuştur.Paroksismal noktürnal hemoglobinüri (PNH) nadir görülen kronik, ilerleyici ve yaşamı tehdit eden multisistemik bir hastalıktır. Tanı konduktan sonraki ilk beş yıl içerisinde mortalite %35 iken on yıldan sonra % 50’ye ulaşmaktadır. PNH’daki progresif morbidite ve mortalitenin altta yatan nedeni kronik hemoliz iken eşlik eden tromboz ölümlerin % 40-67’sinden sorumlu olabilmektedir. Olguların % 40’ı klinik olarak saptanabilen trombotik olaylar yaşar. Tromboz tedavisinde antikoagülan uygulaması yanı sıra eğer kullanılmıyorsa ekulizumab’ın da başlanması önerilmektedir. Ekulizumab tedavisi altında tromboz gelişmesi nadir bir komplikasyondur. Burada kemik iliği yetmezliği ile başvuran, kök hücre nakli (KHN) hazırlık aşamasında ekulizumab tedavisi altında tromboz gelişen ve hızla ilerleyen bir PNH olgusu literatür eşliğinde bildirilmektedir.Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, progressive and life-threatening multisystem disease. Within the first five years after diagnosis, mortality is 35%, but after ten years it reaches 50%. The underlying cause of progressive morbidity and mortality in PNH is chronic hemolysis, which is responsible for 40-67% of accompanying thrombotic deaths. 40% of the cases experience clinically detectable thrombotic events. In addition to anticoagulant therapy in the treatment of thrombosis, it is recommended to start ekulizumab if not used. Development of thrombosis under the treatment of equlizumab is a rare complication. It is reported here that a PNH patient who develops thrombosis under equlizumab therapy and progresses rapidly with stem cell transplantation (SCT) preparation with bone marrow failure

The relation of body iron load and transplantation related complications ın allogenic stem cell transplanted pediatric patients with acute leukemia and myelodysplasia

...The European Group for Blood & Marrow Transplantatio

Nephrotic syndrome after allogeneic hematopoietic stem cell transplantation as a late complication of chronic graft-versus-host disease in three children

...The European Group for Blood & Marrow Transplantatio

A single center experience with allogeneic hematopoietic stem cell transplantation for acquired severe aplastic anaemia in childhood

...European Group for Blood and Marrow Transplantatio

HLA-A allele mismatch (7/8 or 9/10) is the second best option after 8/8 or 10/10 matched unrelated donors: An analysis on results from Turkish centers

Introduction: Hematopoietic stem cell transplantation (HSCT) from an unrelated donor has been established as an effective treatment option for patients with hematological diseases who lack a human leukocyte antigen (HLA)-matched related donor. However, HLA mismatch at the genetic level (allele mismatch) may be observed among serologically HLAmatched (antigen match) donor-recipient pairs, which adversely affects the incidence of severe graft-versus-host disease (GVHD) and survival. The aim of this retrospective multicenter study was to evaluate the impact of HLA mismatch on unrelated transplantation outcomes in Turkey. Materials (or patients) and methods: The data set consisted of follow-up records of 444 (of which 436 with HLA matching data available) unrelated-donor stem cell transplantations performed at 14 centers between July 2002- September 2014 and facilitated by TRAN orTRIS .215 patients underwent single antigen and/or allele-mismatched (mm) HSCT. The distribution of the mismatches according to the HLA-A, HLA-B, HLA-C, and HLA-DR and HLA-DQ loci are:82, 58, 32, 35 and 9 patients, respectively.Twelve patients were transplanted with 8/10 HLA matching. The patients’ characteristics are summarized in Table 1. Results: The neutrophil engraftment was achieved in 82.2% of the patients. HLA mm has a negative impact on engraftment (HLA mm: median 17days vs HLA-matched: median 16 days, p=0.03). Acute GVHD wasobserved at a rate of 42.1%. HLA matching did not have an impact on the incidence of acute GvHD (p=0.35) but chronic GvHD was more frequent among HLA allele /antigen mismatched patients than HLA-matched (p=0.008). The possibility of 5-year overall survival (OS) was 50.2%±2.5%. The presence of HLA mismatch significantly shortened the OS (58.9±3.4% vs Allele mm: 49.8±5.7% vs Antigen mm 36.0±4.9%, p<0.0001).Among the allele level mm HLA-A mm was associated with better OS compared to other loci (55.9±11.7%vs. 17.2±8.2%). When analysis was performed regardless of HLA match or only among HLA matched donor-recipient-pairsgender and stem cell source (PB vs BM) did not have an impact on OS. The OS of patients transplanted between 2002-2007 were shorter than those transplanted later (2008-2014)(37.9±6.4% vs. 52.9±2.7; p= 0.02).European Society for Blood and Marrow Transplantatio