Hematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria with or without Aplastic Anemia: A Multicenter Turkish ExperienceFergün Yılmaz1, Nur Soyer2, Güldane Cengiz Seval3, Sinem Civriz Bozdağ4, Pervin Topcuoğlu3, Ali Ünal5, Leylagül Kaynar6, Gökhan Özgür7, Gülsan Türköz Sucak8, Hakan Göker9, Mustafa Velet9, Hakan Özdoğu10, Mehmet Yılmaz11, Emin Kaya12, Ozan Salim13, Burak Deveci14, İhsan Karadoğan14, Güray Saydam2, Fahri Şahin2, Filiz Vural21Marmara University School of Medicine, Department of Hematology, Istanbul, Turkey;PESG, PNH and Education Study Group
2Ege University School of Medicine, Department of Hematology, Izmir, Turkey;PESG, PNH and Education Study Group
3Ankara University School of Medicine, Department of Hematology, Ankara, Turkey
4Ankara University School of Medicine, Department of Hematology, Ankara, Turkey;PESG, PNH and Education Study Group
5Erciyes University School of Medicine, Department of Hematology, Ankara, Turkey;PESG, PNH and Education Study Group
6Erciyes University School of Medicine, Department of Hematology, Ankara, Turkey
7Medical Park, Bahcesehir Hospital, Hematology and Transplantation Clinic, Istanbul, Turkey; PESG, PNH and Education Study Group
8Medical Park, Bahcesehir Hospital, Hematology and Transplantation Clinic, Istanbul, Turkey
9Hacettepe University School of Medicine, Department of Hematology, Ankara, Turkey
10Baskent University School of Medicine, Department of Hematology, Adana Bone Marrow Transplantation Center, Adana, Turkey
11SANKO University School of Medicine, Department of Hematology, Gaziantep, Turkey;PESG, PNH and Education Study Group
12İnönü University School of Medicine, Department of Hematology, Malatya, Turkey;PESG, PNH and Education Study Group
13Akdeniz University, School of Medicine, Department of Hematology, Antalya, Turkey; PESG, PNH and Education Study Group
14Medstar Antalya Hospital Bone Marrow Transplantation Center / Istanbul Gelisim University, Department of Hematology, Antalya, Turkey
Objective: Although inhibition of the complement system at different steps is a promising therapy modality in patients with paroxysmal nocturnal hemoglobinuria (PNH), allogeneic hematopoietic stem cell transplantation (HCT) is still the only curative therapy, especially for patients with intractable hemolysis or bone marrow failure. The aim of this study is to evaluate the outcomes of allogeneic HCT in PNH patients with aplastic anemia (PNH-AA) or without.
Materials and Methods: Thirty-five PNH/PNH-AA patients who were treated with allogeneic HCT in 10 transplantation centers in Turkey were retrospectively analyzed.
Results: Sixteen (45.7%) and 19 (54.3%) patients were diagnosed with classical PNH and PNH-AA, respectively. The median age of the patients was 32 (18-51) years. The 2-year overall survival (OS) rate and rate of graft-versus-host disease-free, failure-free survival (GFFS) was 81.2% and 78.1%, respectively. The 2-year OS in cases of classical PNH and PNH-AA was 81.3% and 79.9%, respectively (p=0.87), and 2-year GFFS in cases of PNH and PNH-AA was 79% and 76% (p=0.977), without statistical significance. The OS and GFFS rates also did not differ between transplantations with matched sibling donors (MSDs) and matched unrelated donors (MUDs).
Conclusion: Allogeneic HCT with MSDs or MUDs is a good option for selected patients with classical PNH and PNH-AA. In particular, patients with debilitating and refractory hemolysis and patients with bone marrow failure might form an excellent group of candidates for allogeneic HCT.
Keywords: Paroxysmal nocturnal hemoglobinuria, Transplantation, Allogeneic stem cell transplantation, Aplastic anemia
Aplastik Aneminin Eşlik Ettiği ve Etmediği Paroksismal Noktürnal Hemoglobinüri Hastalarında Hematopoetik Kök Hücre Nakli Deneyimi: Çok Merkezli Türkiye DeneyimiFergün Yılmaz1, Nur Soyer2, Güldane Cengiz Seval3, Sinem Civriz Bozdağ4, Pervin Topcuoğlu3, Ali Ünal5, Leylagül Kaynar6, Gökhan Özgür7, Gülsan Türköz Sucak8, Hakan Göker9, Mustafa Velet9, Hakan Özdoğu10, Mehmet Yılmaz11, Emin Kaya12, Ozan Salim13, Burak Deveci14, İhsan Karadoğan14, Güray Saydam2, Fahri Şahin2, Filiz Vural21Marmara University School of Medicine, Department of Hematology, Istanbul, Turkey;PESG, PNH and Education Study Group
2Ege University School of Medicine, Department of Hematology, Izmir, Turkey;PESG, PNH and Education Study Group
3Ankara University School of Medicine, Department of Hematology, Ankara, Turkey
4Ankara University School of Medicine, Department of Hematology, Ankara, Turkey;PESG, PNH and Education Study Group
5Erciyes University School of Medicine, Department of Hematology, Ankara, Turkey;PESG, PNH and Education Study Group
6Erciyes University School of Medicine, Department of Hematology, Ankara, Turkey
7Medical Park, Bahcesehir Hospital, Hematology and Transplantation Clinic, Istanbul, Turkey; PESG, PNH and Education Study Group
8Medical Park, Bahcesehir Hospital, Hematology and Transplantation Clinic, Istanbul, Turkey
9Hacettepe University School of Medicine, Department of Hematology, Ankara, Turkey
10Baskent University School of Medicine, Department of Hematology, Adana Bone Marrow Transplantation Center, Adana, Turkey
11SANKO University School of Medicine, Department of Hematology, Gaziantep, Turkey;PESG, PNH and Education Study Group
12İnönü University School of Medicine, Department of Hematology, Malatya, Turkey;PESG, PNH and Education Study Group
13Akdeniz University, School of Medicine, Department of Hematology, Antalya, Turkey; PESG, PNH and Education Study Group
14Medstar Antalya Hospital Bone Marrow Transplantation Center / Istanbul Gelisim University, Department of Hematology, Antalya, Turkey
Amaç: Paroksismal noktürnal hemoglobunüri (PNH) hastalarının tedavisinde kompleman sisteminin değişik basamaklardaki inhibisyonu umut vadedici bir tedavi yöntemidir. Ancak hastalığın belirti ve bulgularını kontrol etmekte etkin bir tedavi yöntemi olsa da küratif bir tedavi seçeneği değildir. Özellikle kompleman inhibisyonuna rağmen devam eden hemolizi, replasman ihtiyacı ve kemik iliği yetmezliği ile birlikteliği olan hasta grubunda halen tek küratif tedavi seçeneği allojeneik kök hücre naklidir (KHN). Bu çalışmamızda aplastik anemi ile birlikteliği olan (PNH-AA) ve olmayan PNH hastalarında yapılmış allojeneik KHN’nin sonuçlarını değerlendirmeyi amaçladık.
Gereç ve Yöntem: On ayrı merkezde PNH/PNH-AA tanısı olup allojeneik KHN yapılan toplam 35 hasta retrospektif olarak tarandı.
Bulgular: On altı (%45,7) PNH ve 19 (%54,3) PNH-AA hastası çalışmaya dahil edildi. Hastaların ortanca yaşı 32 (18-51) idi. Hastalardaki sağkalım analizlerine baktığımızda; 2 yıllık genel sağkalım (GS) ve graft versus host hastalıksız ve graft yetmezliksiz sağkalım (GFFS) oranları sırasıyla %81,2 ve %78,1 idi. İki yıllık sağkalım PNH ve PNH-AA hastalarında sırasıyla %81,3 ve %79,9 olarak hesaplandı ve gruplar arasında istatistiksel bir fark tespit edilmedi (p=0,87). Tam uyumlu akraba donör ve tam uyumlu akraba dışı donör alt grupları karşılaştırıldığında 2 yıllık OS ve GFFS oranlarında istatistiksel fark gözlenmedi.
Sonuç: Seçilmiş PNH ve PNH-AA hasta grubunda akraba veya akraba dışı tam uyumlu donör ile yapılan allojeneik KHN uygun bir seçenektir.
Özellikle refrakter hemolizi ve replasman ihtiyacı devam eden ve kemik iliği yetmezliği bulguları olan hasta grubunda allojeneik KHN bir tedavi seçeneği olarak değerlendirilmelidir.
Anahtar Kelimeler: Paroksismal noktürnal hemoglobinuri, Transplantasyon, Allojeneik kök hücre nakli, Aplastik anemi
Fergün Yılmaz, Nur Soyer, Güldane Cengiz Seval, Sinem Civriz Bozdağ, Pervin Topcuoğlu, Ali Ünal, Leylagül Kaynar, Gökhan Özgür, Gülsan Türköz Sucak, Hakan Göker, Mustafa Velet, Hakan Özdoğu, Mehmet Yılmaz, Emin Kaya, Ozan Salim, Burak Deveci, İhsan Karadoğan, Güray Saydam, Fahri Şahin, Filiz Vural. Hematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria with or without Aplastic Anemia: A Multicenter Turkish Experience. Turk J Hematol. 2021; 38(3): 195-203
Corresponding Author: Fergün Yılmaz |
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