Successful unrelated donor bone marrow transplantation for paroxysmal nocturnal hemoglobinuria

Woodard, Paul; Wang, W; Pitts, Norman E.; Benaim, E.; Horwitz, Edwin M.; Cunningham, John M.; Bowman, Laura C.

doi:10.1038/sj.bmt.1702827

Cited by 33 publications

(17 citation statements)

References 23 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…Eradication of the PNH clone has been achieved with both myeloablative and reduced-intensity conditioning regimens. [10][11][12][13][14] Younger patients with severe manifestations of the disease (high transfusion requirement, severe pancytopenia, life-threatening thrombosis) and availability of an HLAidentical sibling donor are the best candidates for hematopoietic stem cell transplantation. There are few reports on the use of allogeneic transplantation for paroxysmal nocturnal hemoglobinuria, and nearly all of them include small numbers of patients with only one large survey (57 consecutive patients) reported by the International Bone Marrow Transplant Registry.…”

Section: Introductionmentioning

confidence: 99%

Hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria: long-term results of a retrospective study on behalf of the Gruppo Italiano Trapianto Midollo Osseo (GITMO)

Santarone¹,

Bacigalupo²,

Risitano³

et al. 2009

Haematologica

View full text Add to dashboard Cite

BackgroundParoxysmal 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 MethodsThe 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). ResultsFifteen 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. ConclusionsThe findings of this study confirm that most patients with paroxysmal nocturnal hemoglobinuria may be definitively cured with hematopoietic stem cell transplantation.Key words: paroxysmal nocturnal hemoglobinuria, hematopoietic stem cell transplantation, conditioning regimen. Haematologica 2010;95:983-988. doi:10.3324/haematol.2009 This is an open-access paper. Citation: Santarone S, Bacigalupo A, Risitano AM, Tagliaferri E, Di Bartolomeo E, Iori AP, Rambaldi A, Angelucci E, Spagnoli A, Papineschi F, Tamiazzo S, Di Nicola M, and Di Bartolomeo P. Hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria: long-term results of a retrospective study on behalf of the Gruppo Italiano Trapianto Midollo Osseo (GITMO). Hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria: long-term results of a retrospective study on behalf of the Gruppo Italiano Trapianto Midollo Osseo (GITMO)

show abstract

Section: Introductionmentioning

confidence: 99%

Hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria: long-term results of a retrospective study on behalf of the Gruppo Italiano Trapianto Midollo Osseo (GITMO)

Santarone¹,

Bacigalupo²,

Risitano³

et al. 2009

Haematologica

View full text Add to dashboard Cite

show abstract

“…Based on the lack of spontaneous remissions and poor long-term survival (80 % at 5 years, 60 % at 10 years, and only 28 % at 20 years), HSCT is the recommended treatment of childhood PNH. However, the high prevalence of hemolysis and thrombosis should warrant the consideration of early treatment with anti-complement therapy [33,34].…”

Section: Special Circumstancesmentioning

confidence: 99%

Paroxysmal Nocturnal Hemoglobinuria: From Bench to Bed

Mohammed

EL-Tanni

Atiah

et al. 2016

Indian J Hematol Blood Transfus

View full text Add to dashboard Cite

Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired hemolytic anemia with highly variable clinical symptoms making the diagnosis and prediction of its outcome difficult. It is caused by the expansion of a hematopoietic progenitor cell that has acquired a mutation in the X-linked phosphatidylinositol glycan class A (PIGA) gene that results in deficiency of the glycosylphosphatidylinositol anchor structure responsible for fixing a wide spectrum of proteins particularly CD55 and CD59. The clinical features of this disease arise as a result of complement-mediated hemolysis in unprotected red cells, leukocytes, and platelets as well as the release of free hemoglobin. Patients may present with a variety of clinical manifestations, such as anemia, thrombosis, kidney disease, smooth muscle dystonias, abdominal pain, dyspnea, and extreme fatigue. PNH is an outstanding example of how an increased understanding of pathophysiology may directly improve clinical symptoms and treat disease-associated complications when we inhibit the terminal complement cascade. This topic will discuss PNH overview to assist specialists looking after PNH patients.

show abstract

“…Eradication of the PNH clone has been achieved with both myeloablative and reduced-intensity conditioning regimens (Matozs-Fernandez et al 2009;Suenaga et al 2001). Myeloablative regimens used in PNH consisted of busulfan and cyclophosphamide or fludarabine, high-dose cyclophosphamide alone or together with total body irradiation (TBI) (Raiola et al 2000;Woodard et al 2001). This type of conditioning has been associated with high treatment-related mortality, high-dose cyclophosphamide used alone did not exert a significant effect on PNH clone in the long term (Cho SG et al 2003).…”

Section: Hematopoietic Cell Transplantationmentioning

confidence: 99%

Allogeneic Hematopoietic Cell Transplantation for Paroxysmal Nocturnal Hemoglobinuria

Markiewicz¹,

Koclęga²,

Malgorzata³

et al. 2012

New Advances in Stem Cell Transplantation

View full text Add to dashboard Cite

Successful unrelated donor bone marrow transplantation for paroxysmal nocturnal hemoglobinuria

Cited by 33 publications

References 23 publications

Hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria: long-term results of a retrospective study on behalf of the Gruppo Italiano Trapianto Midollo Osseo (GITMO)

Hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria: long-term results of a retrospective study on behalf of the Gruppo Italiano Trapianto Midollo Osseo (GITMO)

Paroxysmal Nocturnal Hemoglobinuria: From Bench to Bed

Allogeneic Hematopoietic Cell Transplantation for Paroxysmal Nocturnal Hemoglobinuria

Contact Info

Product

Resources

About