Health TrialsbyHealthPanel

Rituximab for Serious Aplastic Anemia With Platelet Transfusion Refractoriness

ConditionsSevere Aplastic AnemiaPlatelet Transfusion Refractoriness
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-70
SponsorInstitute of Hematology & Blood Diseases Hospital, China

About this trial

Due to long-term dependence on platelet transfusion, some severe aplastic anemia (SAA) patients suffer platelet transfusion refractoriness (PTR). Unlike immune thrombocytopenia (ITP), glucocorticoids and human immunoglobulin (IVIg) are generally ineffective for PTR. Due to the lack of effective intervention methods, patients with PTR suffer increased platelet transfusions, bleeding events and treatment costs, prolonged hospital stays, and decreased survival rate. SAA with PTR has become a challenge for physicians. The experiment aims to explore the efficacy of rituximab in the treatment of SAA with PTR, and establish a new effective, safe treatment method with relatively low treatment cost.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Initial diagnosed SAA with PTR

Age>18 years old, regardless of gender

Initial diagnosed SAA with PTR

Age>18 years old, regardless of gender

Disqualifiers

Allergy to rituximab

Severe active infection

Hypogammaglobulinemia

Pregnant and lactating women

Trial design

Design model

Single group

Treatments tested in this trial

  • Rituximab

    Drug

    Rituximab is administered at a dose of 100mg per week, a total of 4 times. Cyclosporin is administered at a dose of 3-5mg/kg per day.

Treatment groups

20 Participants
are divided into 1 treatment group
Group A: rituximab groupExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

The response and complete remission rate with Rituximab protocol.

Response will be evaluated at each clinic visit. Complete response (CR) was defined as achieving all three peripheral blood count criteria: (1) Hb level up to the normal range; (2) ANC≥1.5×109/L; (3) PLT≥100×109/L. Partial response (PR) was defined as transfusion independent, no longer meeting criteria for severe disease. Persistence of transfusion requirement or death was evidence of no response (NR).

Time frame
6 months

Secondary outcomes

1

Relapse rate

Relapse was defined as a responder who met criteria for SAA again after achieving response and keeping stable blood counts for at least 3 months.

Time frame
12 months and 60 months
2

Sustained response (SR)

SR was defined as Hb \> 10 g/dL at 12 months and 60 months, in the absence of any treatment.

Time frame
12 months and 60 months
3

Survival

Survival rate within 5 years after diagnosis

Time frame
60 months
4

Clonal evolution to myelodysplasia and acute leukemia.

Clonal evolution within 5 years after diagnosis

Time frame
60 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Institute of Hematology & Blood Diseases Hospital, China

Lead sponsor

Source ClinicalTrials.gov
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