Combination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer
Allogeneic Blood or Marrow Transplantation for Hematologic Malignancy and Aplastic Anemia
RATIONALE: Giving chemotherapy drugs and total-body irradiation before a donor stem cell helps stop the growth of cancer or abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. It is not yet known which combination chemotherapy regimen is most effective when given before a donor stem cell transplant in treating aplastic anemia or hematologic cancer.
PURPOSE: This phase II/III trial is studying different combination chemotherapy regimens to compare how well they work when given before donor stem cell transplant in treating patients with aplastic anemia or hematologic cancer.
調査の概要
状態
詳細な説明
OBJECTIVES:
- Compare the morbidity, mortality, and overall outcome of patients with severe aplastic anemia or hematologic malignancy treated with standard vs novel conditioning regimens followed by allogeneic stem cell transplantation.
- Examine the influence of donor histocompatibility on outcome by comparing matched/related, mismatched/related (with or without T-cell depletion), and matched/unrelated transplants with stratification for type of preparative regimen.
- Ensure that patients with uncommon diagnoses will be treated in a uniform fashion with the best therapy available.
OUTLINE: Patients are stratified according to risk of relapse (standard-risk: acute leukemia in first complete remission, chronic myelogenous leukemia in first chronic phase, lymphoma in sensitive first relapse or second remission, primary or untreated myelodysplastic syndromes, or untreated severe aplastic anemia vs high-risk: all others).
Patients are assigned to one of the following conditioning regimens based on diagnosis, risk of relapse, and donor relatedness:
- Regimen 1: Patients receive busulfan IV over 2 hours every 6 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
- Regimen 2: Patients receive cyclophosphamide IV over 2 hours on days -5 to -2 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -3.
- Regimen 3: Patients receive cyclophosphamide IV over 2 hours on days -5 and -4 and total-body irradiation (TBI) twice daily on days -3 to -1.
- Regimen 4: Patients receive fludarabine IV over 30 minutes on days -6 to -2 and melphalan IV over 1 hour on days -3 and -2.
- Regimen 5: Patients receive etoposide IV over 26 hours beginning on day -5, cyclophosphamide IV over 2 hours on day -4, and TBI twice daily on days -3 to -1.
- Regimen 6: Patients receive cyclophosphamide IV over 24 hours, carboplatin IV over 24 hours, and thiotepa IV over 24 hours on days -7 to -4.
- Regimen 7: Patients receive fludarabine IV over 30 minutes on days -5 to -1 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -2.
- Regimen 8: Patients receive cyclophosphamide IV over 2 hours on days -5 and -4, TBI twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4-8 hours on days -3 to -1.
- Regimen 9: Patients receive busulfan IV over 2 hours every 6 hours and anti-thymocyte globulin IV over 4-8 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
All patients then receive donor stem cell infusions on day 0. Some patients may undergo involved-field radiotherapy 4-8 weeks after transplant.
Patients will be taken off study after a minimum of 4 years of follow up.
PROJECTED ACCRUAL: At least 405 patients will be accrued for this study within 5 years.
研究の種類
入学 (実際)
段階
- フェーズ2
- フェーズ 3
連絡先と場所
研究場所
-
-
New York
-
Buffalo、New York、アメリカ、14263-0001
- Roswell Park Cancer Institute
-
-
参加基準
適格基準
就学可能な年齢
健康ボランティアの受け入れ
受講資格のある性別
説明
DISEASE CHARACTERISTICS:
Diagnosis of one of the following:
Severe aplastic anemia as defined by either of the following:
- Marrow cellularity (< 25% [or 25-50% cellularity with < 30% of remaining cells hematopoietic in origin])
At least 2 of the following abnormal peripheral blood counts:
- Reticulocyte count < 1% (corrected for hematocrit)
- Platelet count < 20,000/mm^3
- Neutrophil count < 500/mm^3
Histologically confirmed hematologic malignancy, including any of the following:
Acute leukemia
- Resistant or recurrent disease after combination chemotherapy with at least one standard regimen OR in first remission and at high risk of relapse
- Acute myeloid leukemia (AML) (antecedent myelodysplastic syndromes [MDS], secondary AML, or high-risk cytogenetic abnormalities)
- Acute lymphoblastic leukemia (ALL) (high-risk cytogenetic abnormalities)
Chronic myeloid leukemia (CML)
- Chronic phase, accelerated phase, or blast phase
Myeloproliferative disorders or MDS, including any of the following:
- Myelofibrosis
- Polycythemia vera*
- Essential thrombocythemia*
- Refractory anemia
- Refractory anemia with excess blasts
- Refractory anemia with excess blasts in transformation
- Chronic myelomonocytic leukemia NOTE: * Only if transformed to AML or MDS
Lymphoproliferative disease
Recurrent or persistent, symptomatic disease after first-line chemotherapy, including any of the following:
- Chronic lymphocytic leukemia (CLL) (≥ 20% marrow involvement)
- Waldenstrom macroglobulinemia
- Low-grade non-Hodgkin lymphoma
Intermediate or high-grade non-Hodgkin lymphoma, meeting 1 of the following criteria:
- Resistant or recurrent disease after combination chemotherapy with one standard regimen
- Lymphoblastic lymphoma or small noncleaved cell lymphoma in first remission and at high risk of relapse
- CNS disease
- Bone marrow disease and LDH greater than 300
- Solid tumor that would otherwise be treated on RPCI-DS-9115 (or equivalent autologous stem transplant protocol) AND has a syngeneic donor
Autologous bone marrow transplant not possible (or desirable) due to 1 of the following:
- History of marrow tumor
- Inadequate marrow dose
- Abnormal marrow histology or function prior to storage
- Thrombocytopenia or leukopenia
- Marrow cellularity < 20%
Histocompatible donor identified
Well-matched donor, as defined by 1 of the following:
- Family member matched for 5 or 6 HLA specificities (A, B, DR)*
- Unrelated donor meeting compatibility criteria of the National Marrow Donor Program (matched for HLA A, B, and DRB1 antigens)*
- Identical twin sibling
- If a compatible cord blood donor is identified and there is no suitable unrelated donor available, patient may receive cord blood transplant NOTE: *Patients ≤ 25 years of age may be singly mismatched at the A or B loci
NOTE: A new classification scheme for adult non-Hodgkin's lymphoma has been adopted by PDQ. The terminology of "indolent" or "aggressive" lymphoma will replace the former terminology of "low", "intermediate", or "high" grade lymphoma. However, this protocol uses the former terminology.
PATIENT CHARACTERISTICS:
Age:
- 4 to 70
Performance status:
- Zubrod 0-2 OR
- Karnofsky 70-100%
Life expectancy:
- Not specified
Hematopoietic:
- See Disease Characteristics
Hepatic:
- Bilirubin < 3 times normal (unless due to disease)
- Alkaline phosphatase < 3 times normal (unless due to disease)
- SGOT < 3 times normal (unless due to disease)
- Hepatitis B surface antigen negative
- No severe hepatic disease that would preclude study participation
Renal:
- Creatinine normal
- Creatinine clearance ≥ 50 mL/min
- No severe renal disease that would preclude study participation
Cardiovascular:
- Cardiac ventricular ejection fraction ≥ 50% by MUGA or echocardiogram
- No uncontrolled or severe cardiovascular disease (e.g., myocardial infarction, congestive heart failure, symptomatic angina, life threatening arrhythmia, or hypertension within the past 6 months)
Pulmonary:
- DLCO or DLVA ≥ 50% predicted (corrected for hemoglobin or alveolar ventilation)
Other:
- No serious concurrent medical or psychiatric illness
No other serious organ dysfunction (unless due to underlying disease), including the following:
- Uncontrolled bacterial, viral, or fungal infection
- Uncontrolled peptic ulcer disease
- Uncontrolled diabetes mellitus
- HIV negative
- Cytomegalovirus status known
- Not pregnant
PRIOR CONCURRENT THERAPY:
Biologic therapy:
- Not specified
Chemotherapy:
- See Disease Characteristics
- Pretransplant cytoreductive chemotherapy allowed for patients with relapsed or refractory disease
Endocrine therapy:
- Not specified
Radiotherapy:
Not eligible for total-body irradiation if prior radiotherapy exceeded the following limits:
- Mediastinum: 3,600 cGy
- Heart: 3,600 cGy
- Whole lungs: 1,200 cGy
- Small bowel: 3,600 cGy
- Kidneys: 1,200 cGy
- Whole liver: 1,600 cGy
- Cranial spinal: 3,600 cGy
- Brain: 4,000 cGy
- Retina: 4,000 cGy
Surgery:
- Not specified
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:非ランダム化
- 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 |
|---|---|
|
実験的:Regimen 1
Patients receive busulfan IV over 2 hours every 6 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
|
与えられた IV
与えられた IV
|
|
実験的:Regimen 2
Patients receive cyclophosphamide IV over 2 hours on days -5 to -2 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -3.
|
与えられた IV
与えられた IV
|
|
実験的:Regimen 3
Patients receive cyclophosphamide IV over 2 hours on days -5 and -4 and total-body irradiation (TBI) twice daily on days -3 to -1.
|
与えられた IV
Given twice daily for 3 days
|
|
実験的:Regimen 4
Patients receive fludarabine IV over 30 minutes on days -6 to -2 and melphalan IV over 1 hour on days -3 and -2.
|
与えられた IV
与えられた IV
|
|
実験的:Regimen 5
Patients receive etoposide IV over 26 hours beginning on day -5, cyclophosphamide IV over 2 hours on day -4, and TBI twice daily on days -3 to -1.
|
与えられた IV
与えられた IV
Given twice daily for 3 days
|
|
実験的:Regimen 6
Patients receive cyclophosphamide IV over 24 hours, carboplatin IV over 24 hours, and thiotepa IV over 24 hours on days -7 to -4.
|
与えられた IV
与えられた IV
与えられた IV
|
|
実験的:Regimen 7
Patients receive fludarabine IV over 30 minutes on days -5 to -1 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -2.
|
与えられた IV
与えられた IV
|
|
実験的:Regimen 8
Patients receive cyclophosphamide IV over 2 hours on days -5 and -4, TBI twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4-8 hours on days -3 to -1.
|
与えられた IV
与えられた IV
Given twice daily for 3 days
|
|
実験的:Regimen 9
Patients receive busulfan IV over 2 hours every 6 hours and anti-thymocyte globulin IV over 4-8 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
|
与えられた IV
与えられた IV
与えられた IV
|
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 |
|---|---|---|
|
CR Rate
時間枠:day 100
|
Rate of Complete Remission by Day +100
|
day 100
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Toxicity/TRM at Day 100
時間枠:Day +100
|
Death due to treatment related causes before day +100 after BMT
|
Day +100
|
|
4 Year PFS
時間枠:4 years
|
progression free survival estimate at 4 years post BMT (events are disease progression/relapse and death due to any cause)
|
4 years
|
|
4 yr OS
時間枠:4-year
|
Overall survival estimate at 4 years post BMT
|
4-year
|
協力者と研究者
捜査官
- スタディチェア:Philip L. McCarthy, MD、Roswell Park Cancer Institute
研究記録日
主要日程の研究
研究開始 (実際)
一次修了 (実際)
研究の完了 (実際)
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 (見積もり)
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
本研究に関する用語
キーワード
- 詳細不明の小児固形腫瘍、プロトコル固有
- 詳細不明の成人固形腫瘍、プロトコル固有
- 原発性骨髄線維症
- 再発性グレード 3 濾胞性リンパ腫
- 再発性成人びまん性大細胞型リンパ腫
- 再発性成人免疫芽細胞性大細胞型リンパ腫
- 再発成人バーキットリンパ腫
- 再発小児小非分割細胞リンパ腫
- 再発小児大細胞型リンパ腫
- 不応性貧血
- 過剰な芽球を伴う難治性貧血
- 形質転換中の過剰な芽球を伴う難治性貧血
- 慢性骨髄単球性白血病
- de novo 骨髄異形成症候群
- 以前に治療された骨髄異形成症候群
- 二次性骨髄異形成症候群
- 11q23 (MLL) 異常を伴う成人急性骨髄性白血病
- inv(16)(p13;q22)を伴う成人急性骨髄性白血病
- t(15;17)(q22;q12)を伴う成人急性骨髄性白血病
- t(16;16)(p13;q22)を伴う成人急性骨髄性白血病
- t(8;21)(q22;q22)を伴う成人急性骨髄性白血病
- 続発性急性骨髄性白血病
- 寛解期の小児急性リンパ芽球性白血病
- 寛解期の小児急性骨髄性白血病
- 慢性期慢性骨髄性白血病
- 小児慢性骨髄性白血病
- 小児骨髄異形成症候群
- 再発性成人急性骨髄性白血病
- 非定型慢性骨髄性白血病
- 骨髄異形成/骨髄増殖性疾患、分類不能
- 寛解期の成人急性骨髄性白血病
- 再発性成人びまん性小細胞分裂型リンパ腫
- 再発性成人びまん性混合細胞リンパ腫
- 芽球期慢性骨髄性白血病
- 慢性骨髄性白血病の再発
- ワルデンシュトレームマクログロブリン血症
- 再発性グレード 1 濾胞性リンパ腫
- 再発性グレード 2 濾胞性リンパ腫
- 再発辺縁帯リンパ腫
- 再発小リンパ球性リンパ腫
- 節外性辺縁帯 粘膜関連リンパ組織の B 細胞性リンパ腫
- リンパ節辺縁帯B細胞リンパ腫
- 脾辺縁帯リンパ腫
- 再発性成人リンパ芽球性リンパ腫
- 再発性マントル細胞リンパ腫
- 再生不良性貧血
- 難治性慢性リンパ性白血病
- 再発性皮膚 T 細胞非ホジキンリンパ腫
- 成人T細胞白血病/リンパ腫の再発
- 眼内リンパ腫
- 血管免疫芽球性T細胞リンパ腫
- 未分化大細胞型リンパ腫
- 再発性菌状息肉症/セザリー症候群
- 再発性成人急性リンパ芽球性白血病
- 真性赤血球増加症
- 本態性血小板血症
- 前リンパ球性白血病
- 小児急性リンパ芽球性白血病の再発
- 加速期慢性骨髄性白血病
- 寛解期の成人急性リンパ芽球性白血病
- 小児急性骨髄性白血病の再発
- 再発小児リンパ芽球性リンパ腫
- 急性未分化白血病
- T細胞大顆粒リンパ球白血病
- バーキットリンパ腫
追加の関連 MeSH 用語
- 病理学的プロセス
- 免疫系疾患
- 組織型別の新生物
- リンパ増殖性疾患
- リンパ疾患
- 免疫増殖性疾患
- 疾患
- 骨髄疾患
- 血液疾患
- 前がん状態
- 骨髄不全疾患
- 新生物
- リンパ腫
- 症候群
- 骨髄異形成症候群
- 白血病
- 前白血病
- 貧血
- 骨髄増殖性疾患
- 骨髄異形成 - 骨髄増殖性疾患
- 貧血、再生不良
- 薬の生理作用
- 薬理作用の分子機構
- 酵素阻害剤
- 抗リウマチ剤
- 代謝拮抗薬、抗腫瘍薬
- 代謝拮抗剤
- 抗悪性腫瘍薬
- 免疫抑制剤
- 免疫学的要因
- 抗悪性腫瘍薬、アルキル化
- アルキル化剤
- 骨髄破壊的アゴニスト
- 抗悪性腫瘍剤、ファイトジェニック
- トポイソメラーゼ II 阻害剤
- トポイソメラーゼ阻害剤
- シクロホスファミド
- カルボプラチン
- エトポシド
- メルファラン
- フルダラビン
- リン酸フルダラビン
- チオテパ
- ブスルファン
- 抗リンパ球血清
その他の研究ID番号
- RP 98-15 (その他の識別子:Roswell Park Cancer Institute)
この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。