scholarly journals Allogeneic hematopoietic cell transplantation for myelofibrosis: A 10-year experience at single institution

2010 ◽  
Vol 85 (11) ◽  
pp. 904-907 ◽  
Author(s):  
Taiga Nishihori ◽  
Janelle Perkins ◽  
Jongphil Kim ◽  
Teresa Field ◽  
Joseph Pidala ◽  
...  
Blood ◽  
2011 ◽  
Vol 118 (7) ◽  
pp. 1971-1978 ◽  
Author(s):  
Weston P. Miller ◽  
Steven M. Rothman ◽  
David Nascene ◽  
Teresa Kivisto ◽  
Todd E. DeFor ◽  
...  

Abstract Cerebral adrenoleukodystrophy (cALD) remains a devastating neurodegenerative disease; only allogeneic hematopoietic cell transplantation (HCT) has been shown to provide long-term disease stabilization and survival. Sixty boys undergoing HCT for cALD from 2000 to 2009 were analyzed. The median age at HCT was 8.7 years; conditioning regimens and allograft sources varied. At HCT, 50% demonstrated a Loes radiographic severity score ≥ 10, and 62% showed clinical evidence of neurologic dysfunction. A total of 78% (n = 47) are alive at a median 3.7 years after HCT. The estimate of 5-year survival for boys with Loes score < 10 at HCT was 89%, whereas that for boys with Loes score ≥ 10 was 60% (P = .03). The 5-year survival estimate for boys absent of clinical cerebral disease at HCT was 91%, whereas that for boys with neurologic dysfunction was 66% (P = .08). The cumulative incidence of transplantation-related mortality at day 100 was 8%. Post-transplantation progression of neurologic dysfunction depended significantly on the pre-HCT Loes score and clinical neurologic status. We describe the largest single-institution analysis of survival and neurologic function outcomes after HCT in cALD. These trials were registered at www.clinicaltrials.gov as #NCT00176904, #NCT00668564, and #NCT00383448.


2020 ◽  
Vol 40 (11) ◽  
pp. 6531-6537
Author(s):  
KRZYSZTOF CZYŻEWSKI ◽  
ROBERT DĘBSKI ◽  
NATALIA BARTOSZEWICZ ◽  
EWA DEMIDOWICZ ◽  
MONIKA RICHERT-PRZYGOŃSKA ◽  
...  

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