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.jpg): Failed to open stream: No such file or directory in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 117 Biol+Blood+Marrow+Transplant
2014 ; 20
(12
): 1996-2003
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English Wikipedia
Treosulfan-based conditioning and hematopoietic cell transplantation for
nonmalignant diseases: a prospective multicenter trial
#MMPMID25196857
Burroughs LM
; Nemecek ER
; Torgerson TR
; Storer BE
; Talano JA
; Domm J
; Giller RH
; Shimamura A
; Delaney C
; Skoda-Smith S
; Thakar MS
; Baker KS
; Rawlings DJ
; Englund JA
; Flowers ME
; Deeg HJ
; Storb R
; Woolfrey AE
Biol Blood Marrow Transplant
2014[Dec]; 20
(12
): 1996-2003
PMID25196857
show ga
Hematopoietic cell transplantation is an effective treatment for patients with
nonmalignant diseases and for many is the only known cure. Conventional
myeloablative regimens have been associated with unacceptably high early
transplant-related mortality (TRM), particularly in patients with comorbid
conditions. This prospective multicenter trial was designed to determine the
safety and engraftment efficacy of treosulfan-based conditioning in patients with
nonmalignant diseases. Thirty-one patients received HLA-matched related (n = 4)
or unrelated (n = 27) grafts after conditioning with treosulfan (total dose,
42 g/m(2)), fludarabine (total dose, 150 mg/m(2)), ± thymoglobulin (6 mg/kg;
n = 22). Graft-versus-host disease (GVHD) prophylaxis consisted of tacrolimus and
methotrexate. All patients engrafted. Day-100 TRM was 0%. With a median follow-up
of 2 years, the 2-year survival was 90%. Three patients died of GVHD, recurrent
hemophagocytic lymphohistiocytosis, and a surgical complication, respectively.
The cumulative incidences of grades II to IV and III to IV acute GVHD at day 100
and chronic GVHD at 2 years were 62%, 10%, and 21%, respectively. Patients who
received thymoglobulin had a significantly lower incidence of grades III to IV
acute GVHD (0% versus 33%; P = .005). These results indicate that the combination
of treosulfan, fludarabine, and thymoglobulin is effective at establishing donor
engraftment with low toxicity and improved survival in patients with nonmalignant
diseases and support the need for future disease-specific clinical trials.