BACKGROUND: Paroxysmal nocturnal hemoglobinuria is an acquired clonal disorder of the hemopoietic stem cells for which the only curative treatment is allogeneic hematopoietic stem cell transplantation. DESIGN AND METHODS: The aim of this retrospective study was to assess the long-term clinical and hematologic results in 26 paroxysmal nocturnal hemoglobinuria patients who received hematopoietic stem cell transplantation in Italy between 1988 and 2006. The patients were aged 22 to 60 years (median 32 years). Twenty-three donors were HLA-identical (22 siblings and one unrelated) and 3 were HLA-mismatched (2 related and one unrelated). RESULTS: Fifteen patients received a myeloablative conditioning consisting of busulfan and cyclophosphamide (in all cases from identical donor) and 11 were given a reduced intensity conditioning (8 from identical donor and 3 from mismatched donor). The cumulative incidence of graft failure was 8% (4% primary and 4% secondary graft failure). Transplant-related mortality for all patients was 42% (26% and 63% for patients transplanted following myeloablative or reduced intensity conditioning, respectively). As of October 31, 2009, 15 patients (11 in the myeloablative conditioning group and 4 in the reduced intensity conditioning group) are alive with complete hematologic recovery and no evidence of paroxysmal nocturnal hemoglobinuria following a median follow-up of 131 months (range 30-240). The 10-year Kaplan-Meier probability of disease-free survival was 57% for all patients: 65% for 23 patients transplanted from identical donor and 73% for 15 patients transplanted with myeloablative conditioning. No thromboembolic event nor recurrence of the disease were reported following transplant. CONCLUSIONS: The findings of this study confirm that most patients with paroxysmal nocturnal hemoglobinuria may be definitively cured with hematopoietic stem cell transplantation.
BACKGROUND:Paroxysmal nocturnal hemoglobinuria is an acquired clonal disorder of the hemopoietic stem cells for which the only curative treatment is allogeneic hematopoietic stem cell transplantation. DESIGN AND METHODS: The aim of this retrospective study was to assess the long-term clinical and hematologic results in 26 paroxysmal nocturnal hemoglobinuriapatients who received hematopoietic stem cell transplantation in Italy between 1988 and 2006. The patients were aged 22 to 60 years (median 32 years). Twenty-three donors were HLA-identical (22 siblings and one unrelated) and 3 were HLA-mismatched (2 related and one unrelated). RESULTS: Fifteen patients received a myeloablative conditioning consisting of busulfan and cyclophosphamide (in all cases from identical donor) and 11 were given a reduced intensity conditioning (8 from identical donor and 3 from mismatched donor). The cumulative incidence of graft failure was 8% (4% primary and 4% secondary graft failure). Transplant-related mortality for all patients was 42% (26% and 63% for patients transplanted following myeloablative or reduced intensity conditioning, respectively). As of October 31, 2009, 15 patients (11 in the myeloablative conditioning group and 4 in the reduced intensity conditioning group) are alive with complete hematologic recovery and no evidence of paroxysmal nocturnal hemoglobinuria following a median follow-up of 131 months (range 30-240). The 10-year Kaplan-Meier probability of disease-free survival was 57% for all patients: 65% for 23 patients transplanted from identical donor and 73% for 15 patients transplanted with myeloablative conditioning. No thromboembolic event nor recurrence of the disease were reported following transplant. CONCLUSIONS: The findings of this study confirm that most patients with paroxysmal nocturnal hemoglobinuria may be definitively cured with hematopoietic stem cell transplantation.
Authors: K M Sullivan; E Agura; C Anasetti; F Appelbaum; C Badger; S Bearman; K Erickson; M Flowers; J Hansen; T Loughran Journal: Semin Hematol Date: 1991-07 Impact factor: 3.851
Authors: A M Raiola; M T Van Lint; T Lamparelli; F Gualandi; F Benvenuto; O Figari; N Mordini; G Berisso; S Bregante; F Frassoni; A Bacigalupo Journal: Haematologica Date: 2000-01 Impact factor: 9.941
Authors: T Miyata; J Takeda; Y Iida; N Yamada; N Inoue; M Takahashi; K Maeda; T Kitani; T Kinoshita Journal: Science Date: 1993-02-26 Impact factor: 47.728
Authors: U Hegenbart; D Niederwieser; S Forman; E Holler; S Leiblein; L Johnston; W Pönisch; E Epner; R Witherspoon; K Blume; R Storb Journal: Biol Blood Marrow Transplant Date: 2003-11 Impact factor: 5.742
Authors: Peter Hillmen; Claire Hall; Judith C W Marsh; Modupe Elebute; Michael P Bombara; Beth E Petro; Matthew J Cullen; Stephen J Richards; Scott A Rollins; Christopher F Mojcik; Russell P Rother Journal: N Engl J Med Date: 2004-02-05 Impact factor: 91.245
Authors: R A Nash; M S Pepe; R Storb; G Longton; M Pettinger; C Anasetti; F R Appelbaum; R A Bowden; H J Deeg; K Doney Journal: Blood Date: 1992-10-01 Impact factor: 22.113
Authors: Jason P Cooper; Rafic J Farah; Philip A Stevenson; Ted A Gooley; Rainer Storb; Bart L Scott Journal: Biol Blood Marrow Transplant Date: 2019-02-01 Impact factor: 5.742
Authors: Fahri Sahin; Olga Meltem Akay; Mesut Ayer; Mehmet Sinan Dal; Sehmus Ertop; Osman Ilhan; Volkan Karakus; Mehmet Ali Ozcan; Vildan Ozkocaman; Hayri Ozsan; Ozan Salim; Mahmut Tobu; Anil Tombak; Tulin Firatli Tuglular; Mehmet Yilmaz; Ali Unal; Mustafa Nuri Yenerel; Guray Saydam Journal: Am J Blood Res Date: 2016-08-05
Authors: Régis Peffault de Latour; Hubert Schrezenmeier; Andrea Bacigalupo; Didier Blaise; Carmino A de Souza; Stephane Vigouroux; Roelf Willemze; Louis Terriou; Andre Tichelli; Mohamad Mohty; Sophie de Guibert; Judith C Marsh; Jakob Passweg; Jean Yves Mary; Gerard Socié Journal: Haematologica Date: 2012-06-11 Impact factor: 9.941