Successful application of nonmyeloablative transplantation for paroxysmal nocturnal hemoglobinuria

Suenaga, Kohei; Kanda, Yoshinobu; Niiya, Hironari; Nakai, Kunihisa; Saito, Takeshi; Saito, Akiko; Ohnishi, Mutsuko; Takeuchi, Toshio; Tanosaki, Ryuji; Makimoto, Atsushi; Miyawaki, Shuichi; Ohnishi, Toshihiro; Kanai, Sachiyo; Tobinai, Kensei; Takaue, Yoichi; Mineishi, Shin

doi:10.1016/s0301-472x(01)00632-4

Cited by 45 publications

(32 citation statements)

References 10 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…11 However, the risk of treatment-related mortality after SCT is relatively high, with graft-versushost disease (GvHD) accounting for most of the transplant-related deaths. There are a limited number of singlecenter studies on SCT for PNH [12][13][14][15][16][17][18][19][20][21] (reviewed by Parker 1 and Matos-Fernandez 22 ) and only one registry study involving more than 50 patients. 23 The decision to perform a SCT in PNH has usually been deferred until disease progression to recurrent, life-threatening thromboembolism, refractory or transfusion-dependent hemolytic anemia, or development of SAA.…”

Section: Introductionmentioning

confidence: 99%

Allogeneic stem cell transplantation in paroxysmal nocturnal hemoglobinuria

et al. 2012

View full text Add to dashboard Cite

The online version of this article has a Supplementary Appendix. BackgroundIn the era of eculizumab, identifying patients with paroxysmal nocturnal hemoglobinuria who may benefit from allogeneic stem cell transplantation is challenging. Design and MethodsWe describe the characteristics and overall survival of 211 patients transplanted for paroxysmal nocturnal hemoglobinuria in 83 EBMT centers from 1978 to 2007. Next, we conducted a comparison with a cohort of 402 non-transplanted patients with paroxysmal nocturnal hemoglobinuria diagnosed between 1950 and 2005 in 92 French centers. We compared the occurrence of complications (i.e. thromboembolism and aplastic anemia) using either an individual or a stratum-matching procedure. ResultsAfter a median follow-up of 5 years, the 5-year overall survival rate ± standard error (%) was 68±3 in the transplanted group (54±7 in the case of thromboembolism, 69±5 in the case of aplastic anemia without thromboembolism and 86±6 in the case of recurrent hemolytic anemia without thromboembolism or aplastic anemia). Only thromboembolism as the indication for transplantation was associated with worse outcome (P=0.03). We identified 24 pairs of transplanted and non-transplanted patients with thromboembolism for the matched comparison, with worse overall survival for the transplanted patients (hazard ratio=10.0; 95% confidence interval, 1.3-78.1; P=0.007). This was confirmed by the global matching procedure (P=0.03). As regards aplastic anemia without thromboembolism, 30 pairs were identified for the matched comparison. It was not observed that transplanted patients had a significantly worse overall survival (hazard ratio=4.0; 95% confidence interval, 0.9-18.9; P=0.06). A global matching procedure was not feasible. ConclusionsAllogeneic stem cell transplantation is probably not a suitable treatment option for life-threatening thromboembolism in paroxysmal nocturnal hemoglobinuria.

show abstract

Section: Introductionmentioning

confidence: 99%

Allogeneic stem cell transplantation in paroxysmal nocturnal hemoglobinuria

et al. 2012

View full text Add to dashboard Cite

show abstract

“…The use of RIST is also being explored for myelofibrosis, [78][79][80] solid tumors, 5,81,82 aplastic anemia, 83,84 paroxysmal nocturnal hemoglobinuria (PNH), [85][86][87] thalassemia, 88 sickle cell anemia, 88,89 benign nonhematological conditions such as autoimmune diseases. 90,91 Future studies may also attempt to 'target' the kinetics of complete donor T-cell chimerism according to the need for immediate disease control.…”

Section: Discussionmentioning

confidence: 99%

Reduced-intensity allogeneic hematopoietic stem cell transplantation for acute leukemias: ‘what is the best recipe?’

Kassim

Chinratanalab

Ferrara

et al. 2005

Bone Marrow Transplant

View full text Add to dashboard Cite

“…W ostatnich latach pojawiły się doniesienia o skutecznej eradykacji klonu PNH za pomocą kondycjonowania o obniżonej intensywności opartego na fludarabinie [55], z 6-letnim OS na poziomie 87,8% i niewykrywalnymi granulocytami GPI(-) od 30. do 150. doby po allo-HSCT (mediana 100 dni). Może to być opcją u pacjentów z istotnymi chorobami towarzyszącymi, ograniczającymi zastosowanie kondycjonowania mieloablacyjnego, oraz u mło-dych chorych pragnących zachować możliwości rozrodcze [55,57,58]. Z kolei opcją dla pacjentów z PNH kwalifikujących się do procedury allo-HSCT, ale niemających zgodnego dawcy, może być przeprowadzenie transplantacji szpiku od dawcy haploidentycznego z zastosowaniem cyklofosfamidu po przeszczepieniu [59].…”

Section: Przeszczepienie Allogenicznych Krwiotwórczych Komórek Macierunclassified