Investigation on the effectiveness of horseATG + cyclosporine + romiplostim therapy for patients with untreated aplastic anemia (W-JHS AA03)

未提供0 个研究点目标入组 30 人开始时间: 2025年1月12日最近更新: 2025年6月17日

适应症

试验速览

暂无简介。

•Aged >=18 years and <80 years (at time pointof the sig nature of ICF).
•Patients who understand the informed consent and can sign the informed consent form by his/her free will.
•Patients who obey the protocol visit and other rules.
•Good PS (0, 1 and 2)
•Patients with sever aplastic anemia who meet the percentage of cellular component in a bone marrow biopsy specimen is <25% and no fibrosis and no hematological malignancies.
•Not pregnant woman
•Agree to contraception during the study period.

•Treatment history of hATG, rATG, CsA, EPAG, anabolic steroid and allogeneic hematopoietic stem cell transplantation.
•Fulminant form characterized by the neutrophil count of 0/uL at enrollment and that no response to G-CSF treatment for more than or equal to 1 week.
•Patients with chromosomal abnormalities related to MDS that were defined by WHO 2008 diagnostic. Patients with del (13q) alone and -Y alone positive are eligible.
•Patients with >= 10% of dysplasia in >= 1 series of category A and B defined in Classification of dysplasia edited by Working Group for preparation of morphologic diagnostic criteria of refractory anemia (myelodysplastic syndromes)
•Congenital aplastic anemia including Fanconi anemia.
•Patients who received chemotherapy or radiotherapy or patients who developed a cancer within 5 years of entry.
•Patients with uncontrollable infections or diabetes mellitus.
•HBs antigen positive or HBV DNA positive.
•Patients with hepatic, cardiac, or renal dysfunction requiring treatment.
•Patients who are judged by their physician to be unable to participate in this study.

CR or PR achievement rate at week 12 after protocol treatment

时间窗: week 12 after protocol treatment

hematological response rate by NIH criteria partially revised

Type and frequency of chromosomal abnormalities(protocol treatment initiation)
Type and frequency of somatic gene mutations(protocol treatment initiation)
Hematological response rate(week 26, 52 after protocol treatment)
Correlation between hematological response rate and markers(week 12, 26 and 52 after protocol treatment)
Response duration and recurrence rate
Time to response
Appearance of chromosomal abnormalities(week 26 and 52 after protocol treatment)
Appearance of somatic mutated clone(week 12, 26 and 52 after protocol treatment)
Frequency of adverse events

发起方

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