Ruxolitinib as GVHD Prophylaxis in AA Patients

Study Overview
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Brief Summary
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Our study aimed to determine the prophylactic value of ruxolitinib for aGVHD in HSCT recipients of AA.Ruxolitinib was initiated at the beginning of conditioning regimen at 5 twice daily until 3 months post-transplantation. The cumulative incidence of aGVHD within 6 months after HSCT will be the primary observation item.

Official Title
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Ruxolitinib as Graft-versus-Host Disease Prophylaxis for Allogeneic Stem Cell Transplantation in Aplastic Anemia Patients

Conditions
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The Prophylactic Value of Ruxolitinib for aGVHD in HSCT Recipients of AA

Intervention / Treatment
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* Drug: Ruxolitinib 5 MG BID Oral Tablet


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: (1) diagnosed as aplasitic anemia, including V/SAA,CAA and HAAA. (2) Age elder than or equal to 14 years; (3) Informed consent may be signed by themselves. (4) HIV negative, HBV, HCV negative; (5) Informed consent must be signed before the start of the study procedures, and informed consent must be signed by the patient himself or his immediate family. Considering the patient 's condition, if the patient' s signature is unfavorable for disease treatment, the informed consent form should be signed by the legal guardian or the patient 's immediate family member Exclusion Criteria: (1) received a previous allogeneic HSCT; (2) Uncontrolled infection at enrollment; requires mechanical ventilation or is hemodynamically unstable at the time of enrollment; (3) has severe hepatic insufficiency (defined as Child-Pugh Class C; has serum aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 5 xthe upper limit of normal (ULN) or serum total bilirubin > 2.5 x ULN. (4) has end-stage renal impairment with a creatinine clearance less than 10 mL/min. (5) has both moderate hepatic insufficiency AND moderate renal insufficiency; (6) has documented positive results for human immunodeficiency virus antibody (HIVAb), hepatitis C virus antibody (HCV-Ab) with detectable HCV RNA, or hepatitis B surface antigen (HBsAg) within 90 days prior to enrollments; (7) has active solid tumor malignancies with the exception of localized basal cell or squamous cell skin cancer or the condition under treatment (e.g., lymphomas). (8) Suffering from mental disorders or other conditions and unable to cooperate with the requirements of study treatment and monitoring; (9) unable or unwilling to sign the consent form; (10) patients with other special conditions assessed as unqualified by the investigator

Ages Eligible for Study
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Minimum Age: 14 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: treatment arm<br> | Drug: Ruxolitinib 5 MG BID Oral Tablet<br>* ruxolitinib 5mgBID will be administrated at the beginning of the conditioning regimen until 3 months post-transplantation.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| cumulative incidence of aGVHD | cumulative incidence of aGVHD within 6 months post-HSCT | 6 months post-HSCT |