Table 2.
Selected reports of HCT outcomes in patients with myelofibrosis [33]
| Reference | Timeline of HCT |
N | Median age (range), years |
Conditioning regimen |
% of patients with RIC |
NRM | PFS | OS | Comment |
|---|---|---|---|---|---|---|---|---|---|
| Rondelli [34] | NR | 21 | 54 (27–68) | Multiple | 100 | 10% at 1 y | 81% at 2.7 y | 85% at 2.7 y | Extensive cGVHD in 44%; 2 patients needed DLI for 100% donor chimerism; resolution of fibrosis and splenomegaly in majority |
| Kerbauy [35] | NR | 104 | 49 (18–70) | Multiple, Bu/Cy (62%) | 9 | 35% at 5 y | NR | 61% at 5 y | 3 syngeneic donors, 54 of the patients overlapped with a prior report10; targeted Bu improved OS; comorbidity score had impact on survival |
| Patriarca [36] | 1986–2006 | 100 | 49 (21–68) | Multiple, Bu/Cy 50% of full intensity; Thiotepa + Cy in 46% of RIC | 52 | 43% at 3 y | 35% at 3 y | 42% at 3 y | AHCT before 1995; unrelated donor and longer interval from diagnosis predicted worse outcome but not conditioning intensity; relapse at 2 y 41%, progressive decline in NRM over 20 y studied |
| Kroger [9] | 2002–2007 | 103 | 55 (32–68) | Flu-Bu (100%) | 100 | 16% at 1 y | 51% at 5 y | 67% at 5 y | First prospective study in MF, cGVHD in 43%; 12% NRM for fully matched donor AHCT; age > 55 y and HLA mismatch adversely affected OS; JAK2-positive recipients had better EFS and OS; splenectomy increased risk of relapse |
| Ballen [10] | 1989–2002 | 289 | 47 (18–73) | Multiple, Bu/Cy (43%) | 21 | 35% siblings 50% for URD at 5 y | 33% siblings 27% for URD at 5 y | 37% siblings 30% for URD at 5 y | Relapse at 5 y, 32% in sibling and 23% in URD; performance status, peripheral blasts sibling donor status impacted OS; RIC was similar in outcomes, except early NRM |
| Alchalby [31] | 1999–2009 | 162 | 56 (32–73) | Flu-Bu in 96% | 100 | 22% at 1 y | 46% at 5 y | 62% at 5 y | 82 patients reported previously54; age and HLA mismatch impacted NRM; 23% relapse at 3 y; clearance of mutated JAK2 at median of 96 days, and this reduced relapse risk |
| Bacigalupo [37] | 1994–2007 | 46 | 51 (24–67) | Thiotepa-Cy + melphalan | 100 | 24% at 5 y | NR | 45% at 5 y | A risk score based on transfusion history, spleen > 22 cm and alternative donor use predicted lower OS; no benefit for splenectomy |
| Robin [38] | 1997–2008 | 147 | 53 (20–68) | Multiple | 69 | 39% at 4 y | 32% at 4 y | 39% at 4 y | 19% patients had LT; poor outcome with mismatched donor |
| Samuelson [20] | 1999–2007 | 30 | 65 (60–78) | Multiple | 63 | 13% at day 100 | 40% at 3 y | 45% at 3 y | Studied outcomes in patients ≥ 60 y, 7 patients had preceding LT |
| Ditschkowski [13] | 1994–2010 | 76 | 50.5 (22–67) | Multiple | NR | 36% at 5 y | 50% at 5 y | 53% at 5 y | Significant high risk of relapse in patients without cGvHD; DIPSS was predictive of survival |
| Scott [16] | 1990–2009 | 170 | 51.5 (12–78) | Multiple | NR | 34% at 5 y | 57% at 5 y | 57% at 5 y | Post-HCT success was dependent on pre-HCT DIPSS scores |
MRD=HLA matched related donor; PFS = progression-free survival; OS = overall survival; TBI=, total body irradiation; Hb = hemoglobin; Bu= busulfan; Cy = cyclophosphamide; cGvHD= chronic GVHD; NR= not reported; DLI= donor lymphocyte infusion; AHCT= allogeneic HCT; PFS= event-free survival; NRM= non-relapse mortality; OS= overall survival; KPS= Karnofsky performance status; ATG= antithymocyte globulin.