title: Reduced-intensity stem cell allografting for PNH patients in the eculizumab era: The Mexican experience.
abstract: Background Paroxysmal nocturnal haemoglobinuria (PNH) presents as two major entities: the classical form, predominantly haemolytic and a secondary type with marrow failure and resultant aplastic anaemia (AA-PNH). Currently, the treatment of choice of the haemolytic variant is eculizumab; however, the most frequent form of PNH in México is AA-PNH. Patients and methods Six consecutive AA-PNH patients with HLA-identical siblings were allografted in two institutions in México, employing a reduced-intensity conditioning regimen for stem cell transplantation (RIST) conducted on an outpatient basis. Results Median age of the patients was 37 years (range 25-48). The patients were given a median of 5.4 × 10(6)/kg allogeneic CD34(+) cells, using 1-3 apheresis procedures. Median time to achieve above 0.5 × 10(9)/l granulocytes was 21 days, whereas median time to achieve above 20 × 10(9)/l platelets was 17 days. Five patients are alive for 330-3150 days (median 1437) after the allograft. The 3150-day overall survival is 83.3%, whereas median survival has not been reached, being above 3150 days. Conclusion We have shown that hypoplastic PNH patients can be allografted safely using RIST and that the long-term results are adequate, the cost-benefit ratio of this treatment being reasonable. Additional studies are needed to confirm the usefulness of RIST in the treatment of AA-PNH.
authors.forename: Alejandro
authors.initials: A
authors.lastname: Schcolnik-Cabrera
authors.forename: Nancy
authors.initials: N
authors.lastname: Labastida-Mercado
authors.forename: Laura Samantha
authors.initials: LS
authors.lastname: Galindo-Becerra
authors.forename: David
authors.initials: D
authors.lastname: Gomez-Almaguer
authors.forename: Miguel Angel
authors.initials: MA
authors.lastname: Herrera-Rojas
authors.forename: Guillermo Jose
authors.initials: GJ
authors.lastname: Ruiz-Delgado
authors.forename: Guillermo José
authors.initials: GJ
authors.lastname: Ruiz-Arguelles
mesh_terms: D000328:Adult; D005260:Female; D006086:Graft vs Host Disease / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006457:Hemoglobinuria, Paroxysmal / Q000175:diagnosis / Q000401:mortality / Q000628:therapy*; D006801:Humans; D008297:Male; D008875:Middle Aged; D014019:Tissue Donors; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D014430:Twins, Monozygotic
keywords: Aplastic anaemia; BMT; PNH; Reduced intensity; Stem cell transplant