ALLO-HCT FOR PAROXYSMAL NOCTURNAL HEMOGLOBINURIA-12 YEARS OF EXPERIENCE
PBN-AR
Instytucja
Instytut Hematologii i Transfuzjologii
Informacje podstawowe
Główny język publikacji
en
Czasopismo
Haematologica-The Hematology Journal (40pkt w roku publikacji)
ISSN
0390-6078
EISSN
1592-8721
Wydawca
FERRATA STORTI FOUNDATION
DOI
URL
Rok publikacji
2017
Numer zeszytu
suppl. 2
Strony od-do
300-300
Numer tomu
102
Identyfikator DOI
Liczba arkuszy
Autorzy
(liczba autorów: 7)
Pozostali autorzy
+ 6
Autorzy przekładu
(liczba autorów przekładu: 0)
Konferencja
Indeksowana w Scopus
nie
Indeksowana w Web of Science Core Collection
tak
Liczba cytowań z Web of Science Core Collection
Nazwa konferencji (skrócona)
Nazwa konferencji
22nd Congress Of The European Hematology Association
Początek konferencji
2017-06-22
Koniec konferencji
2017-06-25
Lokalizacja konferencji
Madrid
Kraj konferencji
ES
Lista innych baz czasopism i abstraktów w których była indeksowana
Streszczenia
Język
en
Treść
Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired clonal abnormality of hematopoietic stem cell leading to lack of phosphatidylinositol glycoproteins, sensitizing cells to complement-mediated lysis. Despite the efficient symptomatic treatment of hemolytic PNH with eculizumab, alloHCT is the only curative treatment for the disease, although outcomes presented in the past were controversial. Aims: The aim of this study was to evaluate the safety and effectiveness of allo-HCT in PNH. Methods: We report 41 allo-HCTs: 37 from MUD and 4 from MRD performed for PNH in 2004-2016. Median age of recipients was 29(20-62) years and donors 30(19-53), median time from diagnosis to allo-HCT was 16(2-307) months. Median size of PNH clone was 80% granulocytes (0.5%>100%). Indication for allo-HCT was PNH with aplastic/hypoplastic bone marrow (19 pts), MDS (2 pts), overlapping MDS/aplasia (3 pts), severe course of PNH with hemolytic crises and transfusion-dependency without access to eculizumab (17 pts). Additional risk factors were Budd-Chiari syndrome and hepatosplenomegaly (1 pt), history of renal insufficiency requiring hemodialyses (2 pts), chronic hepatitis B (1 pt) and C (1 pt). The preparative regimen consisted of treosulfan 3x14 g/m2 plus fludarabine 5x30mg/m2 (31 pts) or treosulfan 2x10 g/m2 plus cyclophosphamide 4x40mg/kg (10 pts). Standard GVHD prophylaxis consisted of cyclosporine-A, methotrexate and pre-transplant ATG in MUD-HCT. 2 pts instead of cyclosporine-A received mycophenolate mofetil and tacrolimus. Source of cells was bone marrow (13 pts) or peripheral blood (28 pts) with median 6.3x108NC/kg, 5.7x106CD34+cells/kg, 24.7x107CD3+cells/kg. Myeloablation was complete in all pts with median 9(1- 20) days of absolute agranulocytosis <0.1 G/l. Median number of transfused RBC and platelets units was 9(0-16) and 8(2-18). Results: All pts engrafted, median counts of granulocytes 0.5 G/l, platelets 50 G/l and Hb 10 g/dl were achieved on days 17.5(10-33), 16(9-39) and 19.5(11- 34). Acute GVHD grade I,II and III was present in 16, 7 and 3 pt, limited and extensive chronic GVHD respectively in 11 and 3 pts. LDH decreased by 73%(5%>91%) in first 30 days indicating disappearance of hemolysis. 100% donor chimerism was achieved in all pts. In 1 patient donor chimerism decreased to 81% what was treated with donor lymphocytes infusion (DLI). 3 patients died, 1 previously hemodialysed pt died on day +102 due to nephrotoxicity complicating adenoviral/CMV hemorrhagic cystitis, two other SAA patients with PNH clone<10% died on days +56 due to severe pulmonary infection and +114 due to aGvHD-III and multi organ failure. Complications in survivors were FUO (10 pts), CMV reactivation (13), VOD (1), neurotoxicity (1), venal thrombosis (1), hemorrhagic cystitis (4) and mucositis (8). 38 pts (92.7%) are alive 4.2 (0.4-12) years post-transplant and are doing well without treatment. Complete disappearance of PNH clone was confirmed by flow cytometry in all surviving pts. Summary/Conclusions: We conclude that allo-HCT with treosulfan-based conditioning is effective and well tolerated curative therapy for PNH.
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PX-5971e7abd5dead23f7fbfd01