Allo-HSCT Sonrası İdame Tedavisi Olarak Menin İnhibitörlerinin Etkinliği ve Güvenliği Üzerine Gerçek Yaşam Çalışması

Sade dilde özet

Bu gerçek yaşam çalışması, menin inhibitörlerinin (örneğin Revumenib, Ziftomenib) allojenik kök hücre nakli (allogeneic hematopoietic stem cell transplantation) sonrası idame tedavisi olarak kullanımının etkinliğini ve güvenliğini değerlendirmektedir. Çalışmaya, akut lösemi (acute leukemia) tanısı olan, NPM1 mutasyonu ya da KMT2A/NUP98 gen değişiklikleri bulunan, nakil sonrası menin inhibitörü idame tedavisi almış 15 yaş ve üzeri hastalar alınmaktadır. Faz uygulanmayan bir çalışma olarak yürütülmektedir ve halen katılımcı almaktadır.

Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.

Deney bilgileri

Faz

N/A

Durum

Katılımcı alıyor

Sponsor

The First Affiliated Hospital of Soochow University

Başlangıç

01.11.2025

Tahmini bitiş

31.12.2029

Ülkeler

China

İlgili genler

Merkezler

• Suzhou, China

Uygunluk kriterleri

Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.

Inclusion Criteria:

1. Age ≥ 15 years.
2. Patients diagnosed with acute leukemia (including AML, ALL, and MPAL) according to the World Health Organization (WHO 2022) criteria.
3. Must meet one of the following characteristics: a. Harboring an NPM1 gene mutation (without concurrent FLT3-ITD or FLT3-TKD mutation); b. Harboring a KMT2A gene rearrangement or KMT2A-PTD; c. Harboring a NUP98 gene rearrangement; d. Other acute leukemia subtypes dependent on the menin-KMT2A interaction, if evidenced, may be enrolled upon discussion with and approval from the principal research team.
4. Has undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT), with ≥ 30 days elapsed since the date of graft infusion.
5. Received menin inhibitor maintenance therapy after allo-HSCT and meets the following conditions: a. Received at least ≥ 2 complete cycles (7 days per cycle) of menin inhibitor therapy, or cumulative medication duration ≥ 14 days; b. Patient was in a state of CR/CRh/CRi at the initiation of maintenance therapy.
6. No evidence of leukemia relapse during menin inhibitor maintenance therapy, defined as: a. Bone marrow blasts \< 5%, and blasts do not exhibit morphological features of acute leukemia (e.g.Auer rods); b. No evidence of extramedullary leukemia (e.g. CNS leukemia or myeloid sarcoma).
7. The menin inhibitors used include but are not limited to: Revumenib, BN104, Ziftomenib, HMPL-506, or other menin-KMT2A interaction inhibitors.
8. Capable of understanding and voluntarily signing the informed consent form.
9. Complete clinical data.

Exclusion Criteria:

1. Presence of any of the following at the initiation of menin inhibitor maintenance therapy (including within 28 days prior to starting treatment): a. Morphologic relapse in bone marrow (bone marrow blasts ≥ 5%); b. Presence of leukemic cells in peripheral blood.
2. Active infection that is deemed uncontrolled by the investigator.
3. Severe organ dysfunction, including: a. Hepatic impairment: ALT or AST ≥ 5 × ULN (Upper Limit of Normal), or total bilirubin ≥ 3 × ULN; b. Severe renal impairment: eGFR \< 30 ml/min/1.73 m²; c. Cardiac dysfunction: NYHA (New York Heart Association) Class III-IV.
4. Concurrent acute graft-versus-host disease (aGVHD) ≥ Grade 2 or chronic graft-versus-host disease (cGVHD) ≥ Grade 3, requiring corticosteroids ≥ 1 mg/kg and ≥ 3 types of immunosuppressive therapy (including CNI, ruxolitinib, belumosudil, etc.).
5. History of other malignancies requiring ongoing treatment (except for malignancies that have undergone curative treatment or are assessed to be in complete remission and require no systemic maintenance therapy or radiotherapy).
6. Any gastrointestinal disorder that may affect the intake or absorption of oral medications (e.g.dysphagia, gastroparesis, uncontrolled chronic diarrhea, intestinal GVHD, etc).
7. Patients deemed unsuitable for inclusion in this study by the investigator.
8. Severely missing clinical data, precluding efficacy or safety assessment.
9. Receipt of other maintenance therapies, including but not limited to hypomethylating agents, donor lymphocyte infusion (DLI), or other specific small-molecule targeted drugs.