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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.
During the treatment period, Rituximab is administered at a dose of 100mg per week, a total of 4 times.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| rituximab group | Experimental | Rituximab combined with cyclosporine |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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). | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 12 months and 60 months |
| Sustained response (SR) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Huang Jinbo, MD. | Contact | +86 22 23909023 | huangjinbo@ihcams.ac.cn |
| Name | Affiliation | Role |
|---|---|---|
| Huang Jinbo, MD. | Chinese Academy of Medical Sciences | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences | Recruiting | Tianjin | Tianjin Municipality | 300020 | China |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 26568159 | Background | Killick SB, Bown N, Cavenagh J, Dokal I, Foukaneli T, Hill A, Hillmen P, Ireland R, Kulasekararaj A, Mufti G, Snowden JA, Samarasinghe S, Wood A, Marsh JC; British Society for Standards in Haematology. Guidelines for the diagnosis and management of adult aplastic anaemia. Br J Haematol. 2016 Jan;172(2):187-207. doi: 10.1111/bjh.13853. Epub 2015 Nov 16. No abstract available. | |
| 17667077 |
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| ID | Term |
|---|---|
| D000741 | Anemia, Aplastic |
| ID | Term |
|---|---|
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000080983 | Bone Marrow Failure Disorders |
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| ID | Term |
|---|---|
| D000069283 | Rituximab |
| ID | Term |
|---|---|
| D058846 | Antibodies, Monoclonal, Murine-Derived |
| D000911 | Antibodies, Monoclonal |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
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|
SR was defined as Hb > 10 g/dL at 12 months and 60 months, in the absence of any treatment.
| 12 months and 60 months |
| Survival | Survival rate within 5 years after diagnosis | 60 months |
| Clonal evolution to myelodysplasia and acute leukemia. | Clonal evolution within 5 years after diagnosis | 60 months |
| Background |
| Chockalingam P, Sacher RA. Management of patients refractory to platelet transfusion. J Infus Nurs. 2007 Jul-Aug;30(4):220-5. doi: 10.1097/01.NAN.0000281531.97183.c0. |
| 18564396 | Background | Kerkhoffs JL, Eikenboom JC, van de Watering LM, van Wordragen-Vlaswinkel RJ, Wijermans PW, Brand A. The clinical impact of platelet refractoriness: correlation with bleeding and survival. Transfusion. 2008 Sep;48(9):1959-65. doi: 10.1111/j.1537-2995.2008.01799.x. Epub 2008 Jun 28. |
| D001855 | Bone Marrow Diseases |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |