Classic PNH and PNH with bone marrow failure
Conditions
Interventions
Classic PNH and PNH with bone marrow failure:Pretreatment scheme for full-sib compatibility:
C5 McAb 600 mg for -9 days.Fludarabine (FLU) 30mg/m2/d × 6d (-7 to -2D) + CY 50mg/kg/d × 2D (-4 to -3D) + A
Classic PNH and PNH with bone marrow failure:Alternative donor (unrelated donor, haplotype donor) conditioning regimen:
C5 McAb 600 mg for -9 days.Busulfan (BU) 3.2 mg/kg/d (0.8 mg/kg, q6h) × 2D (-7 t
Classic PNH and PNH with bone marrow failure:Treatment regimen for GVHD: Grade II treatment, methylprednisolone 1 ~ 2mg/kg, divided into two doses, second-line drugs can be selected as appropriate: FK
Classic PNH and PNH with bone marrow failure:Symptomatic and supportive treatment: Transplant-related complications such as infection, bleeding, hemorrhagic cystitis, epilepsy and mucositis, as well a
Classic PNH and PNH with bone marrow failure:Other concomitant therapy: For hemoglobin < 60g/L or red blood cell transfusion therapy is allowed in emergency.Hormones are allowed to control acute hemol
Sponsors
Hongci Hematology Branch of the First Affiliated Hospital of Soochow University
Eligibility
Sex/Gender
All
Age
14 Years to 50 Years
Inclusion criteria
Inclusion criteria: 1) PNH with a definitive diagnosis of classic PNH or PNH with bone marrow failure diseases such as MDS or AA, according to the PNH International Working Group; 2) The age is = 14 years old and less than 50 years old (the age of patients who are ineffective in other treatments and have good physical condition is relaxed to 60 years old). If the patient is not over 14 years old, the applicable informed consent shall be signed according to the age of the patient, different research centers and national standards; 3) No contraindication to hematopoietic stem cell transplantation; 4) Patients and their clients have the desire and request for hematopoietic stem cell transplantation; 5) having a relative donor or substitute donor of blood-providing hematopoietic stem cells; 6) The patient has the ability to understand and is willing to participate in the study, signs the informed consent form (ICF form), and is willing to comply with all study visits and procedures.
Exclusion criteria
Exclusion criteria: 1) Concomitant with other serious physical or mental illnesses that may affect the expression of the condition and the validity of the informed consent or lead to the patient's survival within 2 years; 2) Chronic infections (including bacterial, fungal or viral infections) that are difficult to control, such as dental caries, otitis media and sinusitis, should be carried out after effective control; 3) severe organ dysfunction; 4) decompensated hepatic insufficiency and active hepatitis; 5) History of severe autoimmune disease; 6) Other malignant tumors within 5 years; 7) History of severe allergy to biological products (including antibiotics); 8) Pregnant or lactating women.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Overall survival at 1 and 2 years after transplantation;Failure-free survival;Recurrence rate;Non-relapse-related mortality; | — |
Secondary
| Measure | Time frame |
|---|---|
| Change in fatigue (Functional Assessment of Chronic Disease Therapy [FACIT]), prespecified exploratory endpoints; fatigue scores and QoL (EORTC QLQ-C30) before and after treatment at 1, 2, 3, 6, 12, and 2 years;Hematopoietic reconstitution, engraftment, GVHD incidence, site and grade;Incidence of infection, TA-TMA and SOS; | — |
Countries
China
Contacts
Public ContactLimin Liu
Hongci Hematology Branch of the First Affiliated Hospital of Soochow University()
Outcome results
None listed