A number of groups have demonstrated very low incidence of acute and chronic graft-versus-host disease (GVHD) with post-transplantation cyclophosphamide (PTCy) in haploidentical and unrelated allogeneic stem cell transplantation (SCT). Still the relapse of the underlining malignancy is a problem after this prophylaxis. Ruxolitinib is currently one of the most promising drugs in the treatment of steroid-refractory GVHD. On the other hand, its primary indication is myelofibrosis, and it was demonstrated that ruxolitinib before allogeneic SCT might improve the outcome. This pilot trial evaluates whether the combination of PTCy and ruxolitinib facilitates adequate GVHD control, and decreases the risk of graft failure and disease progression in myelofibrosis patients.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Day 0: Infusion of unmanipulated graft
Days -5 through -3: Busulfan 1 mg/kg po qid №10
Days -7 through -2: 30 mg/m2/day iv qd x 6 days
Day +3 and +4: 50 mg/kg/day iv qd
Days -8 through -2 15 mg tid
Days +5 through +100: 7.5 mg bid
First Pavlov State Medical University of St. Petersburg
Saint Petersburg, Russia
Incidence of acute graft-versus-host disease, grades II-IV
Time frame: 180 days
Incidence of chronic GVHD, moderate and severe (NIH criteria)
Time frame: 365 days
Incidence of primary or secondary graft failure
Time frame: 60 days
Non-relapse mortality analysis
Non-relapse mortality is defined as any death in absence of relapse or progressive disease. Summarized using Kaplan-Meier and cumulative incidence estimates.
Time frame: 365 days
Overall survival analysis
Summarized using Kaplan-Meier and cumulative incidence estimates.
Time frame: 365 days
Event-free survival analysis
Event is defined as relapse or death in the specified time frame. Summarized using Kaplan-Meier and cumulative incidence estimates.
Time frame: 365 days
Relapse rate analysis
Summarized using Kaplan-Meier and cumulative incidence estimates.
Time frame: 365 days
Number of participants with treatment-related adverse events as assessed by CTCAE v4.03
Toxicity parameters based on NCI CTCAE 4.03 grades: hepatotoxicity (liver function tests), nephrotoxicity (creatinine), neurotoxicity (attending physician assessment), mucositis (attending physician assessment), hemorrhagic cystitis (attending physician assessment), cardiotoxicity (ECG, echocardiography). Additional toxicity parameters: incidence and severity of veno-occlusive disease, incidence of transplant-associated microangiopathy
Time frame: 100 days
Infectious complications, including analysis of severe bacterial, fungal and viral infections incidence
Time frame: 100 days
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