Peripheral Blood Versus Bone Marrow Plus Peripheral Blood Grafts for Haploidentical Transplantation in Severe Aplastic Anemia (PB-Graft Haplo)
A Randomized Controlled Trial Comparing Different Graft Sources for Haploidentical Transplantation in Aplastic Anemia
Severe aplastic anemia is a life-threatening bone marrow failure disorder. Haploidentical hematopoietic stem cell transplantation has become an important curative treatment option for patients who do not have an HLA-matched sibling donor.
Traditionally, haploidentical transplantation for severe aplastic anemia uses a graft composed of granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells. However, bone marrow collection is invasive and may increase donor burden. Peripheral blood stem cell collection is simpler and less invasive, but it remains unclear whether using peripheral blood stem cells alone provides similar clinical outcomes without increasing the risk of graft-versus-host disease.
This multicenter, randomized, open-label, non-inferiority trial will compare granulocyte colony-stimulating factor-primed peripheral blood stem cells alone with granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells as graft sources for haploidentical transplantation in patients with severe or very severe aplastic anemia. Participants will be randomly assigned in a 1:1 ratio to either graft source group. The primary outcome is the cumulative incidence of grade II-IV acute graft-versus-host disease within 100 days after transplantation. Secondary outcomes include engraftment, chronic graft-versus-host disease, infections, immune reconstitution, survival, and donor safety.
調査の概要
状態
研究の種類
入学 (推定)
段階
- 適用できない
参加基準
適格基準
就学可能な年齢
- 大人
健康ボランティアの受け入れ
説明
Inclusion Criteria:
Patients with newly diagnosed or relapsed severe aplastic anemia (SAA) or very severe aplastic anemia (vSAA) according to the Camitta criteria. SAA is defined as bone marrow cellularity <25% and at least two of the following peripheral blood criteria: absolute neutrophil count <0.5 × 10⁹/L, platelet count <20 × 10⁹/L, or reticulocyte count <20 × 10⁹/L. vSAA is defined as SAA with an absolute neutrophil count <0.2 × 10⁹/L.
Age 18 to 40 years. Availability of a haploidentical related donor. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.
Adequate organ function, defined as:
Cardiac function: left ventricular ejection fraction ≥50% and no severe arrhythmia; Hepatic function: total bilirubin ≤2 times the upper limit of normal and alanine aminotransferase/aspartate aminotransferase ≤3 times the upper limit of normal; Renal function: creatinine clearance ≥60 mL/min. No active infection. For women of childbearing potential, a negative pregnancy test is required. All patients must agree to use effective contraception during the study period.
Written informed consent provided by the patient or legal representative.
Exclusion Criteria:
Presence of hematologic malignancy or myelodysplastic syndrome-related chromosomal abnormalities, such as +8 or del(7q).
Prior allogeneic hematopoietic stem cell transplantation or prior organ transplantation.
Availability of an HLA-matched sibling donor. Active uncontrolled infection, including unresolved invasive fungal disease, active tuberculosis, sepsis, or other uncontrolled infections.
Severe comorbidities, defined as Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) score ≥4, or uncontrolled autoimmune disease.
Donor-related contraindications, including donor age <12 years or >65 years, donor body mass index <18 or >35 kg/m², contraindications to anesthesia, or contraindications to bone marrow harvest.
Psychiatric illness that prevents cooperation with the study procedures, or any condition considered by the investigator to make the patient unsuitable for study participation.
研究計画
研究はどのように設計されていますか？
デザインの詳細
- 主な目的：処理
- 割り当て：ランダム化
- 介入モデル：並列代入
- マスキング：なし（オープンラベル）
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
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実験的：G-CSF-Primed Peripheral Blood Stem Cell Graft
Participants assigned to this arm will undergo haploidentical hematopoietic stem cell transplantation using granulocyte colony-stimulating factor-primed peripheral blood stem cells alone as the graft source.
Donors will receive granulocyte colony-stimulating factor mobilization, and peripheral blood stem cells will be collected by apheresis.
Participants will receive the protocol-specified conditioning regimen and graft-versus-host disease prophylaxis.
|
Haploidentical hematopoietic stem cell transplantation using granulocyte colony-stimulating factor-primed peripheral blood stem cells alone as the graft source.
Donors will receive granulocyte colony-stimulating factor mobilization, followed by peripheral blood stem cell collection by apheresis.
|
|
実験的：G-CSF-Primed Bone Marrow Plus Peripheral Blood Stem Cell Graft
Participants assigned to this arm will undergo haploidentical hematopoietic stem cell transplantation using granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells as the graft source.
Donors will receive granulocyte colony-stimulating factor mobilization, followed by bone marrow harvest and peripheral blood stem cell collection.
Participants will receive the protocol-specified conditioning regimen and graft-versus-host disease prophylaxis.
|
Haploidentical hematopoietic stem cell transplantation using granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells as the graft source.
Donors will receive granulocyte colony-stimulating factor mobilization, followed by bone marrow harvest and peripheral blood stem cell collection.
|
この研究は何を測定していますか？
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Cumulative Incidence of Grade II-IV Acute Graft-Versus-Host Disease Within 100 Days After Transplantation
時間枠：Up to 100 Days After Transplantation
|
The cumulative incidence of newly diagnosed or persistent grade II-IV acute graft-versus-host disease within 100 days after haploidentical hematopoietic stem cell transplantation.
Acute graft-versus-host disease will be graded according to the MAGIC criteria and assessed based on skin, gastrointestinal, and liver manifestations.
Endpoint adjudication will be performed by an independent blinded endpoint review committee using medical records, photographs, endoscopy reports, and/or biopsy reports, as applicable.
|
Up to 100 Days After Transplantation
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協力者と研究者
研究記録日
主要日程の研究
研究開始 (推定)
一次修了 (推定)
研究の完了 (推定)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
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