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a Clinical Trial of Efficacy and Safety of the Holistic Treatment of Young High-risk Multiple Myeloma Patients

Not Applicable
Conditions
Multiple Myeloma
Plasma Cell Leukemia
Extramedullary Plasmacytoma
Loss of Chromosome 17p
t(14;16)
t(4;14)
T(14;20)
1Q21 Amplification
Complex Karyotype
Interventions
Procedure: Allogeneic Hematopoietic Stem Cell Transplantation
Procedure: Autologous Hematopoietic Stem Cell Transplantation x 1 or x 2
Drug: PI+IMids+Dexamethasone as Consolidated Chemotherapy
Registration Number
NCT04008888
Lead Sponsor
Institute of Hematology & Blood Diseases Hospital, China
Brief Summary

The clinical trial was conducted in a cohort of young, high-risk myeloma patients who were designed to receive a combination of high-dose chemotherapy with allogeneic or autologous hematopoietic stem cell transplantation. The objective was to assess the progression free survival (PFS), overall survival (OS),and overall response rate (ORR) of the overall treatment.

Detailed Description

50 cases of HR-NDMM patients were divided into two groups nonrandomizedly. TE group received hematopoietic stem cell transplantation after induction therapy. Allo-sct for the young patients with suitable donors, Asct for the others. TNE group received consolidation therapy after induction therapy. All patients received PI-based maintenance therapy.

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
50
Inclusion Criteria
  1. Clinical diagnosis of high-risk multiple myeloma

    In addition, patients must meet at least one of the following criteria I-IX (I-VIII at time of diagnosis or pre-autograft):

    I.Complex karyotype

    II.Fluorescent in situ hybridization (FISH) translocation 4:14 or 14:16,

    III.FISH translocation 1q21,

    IV.FISH deletion 17p,

    V.R-ISS III stage,

    VI.Two or more high-risk cytogenetic abnormalities exist

    VII.Plasma cell leukemia

    VIII.Extramedullary plasmacytoma

    IX.Recurrent or non-responsive (less than partial remission [PR]) MM after at least 4 cycles of PI/IMids-based chemotherapy

  2. candidate for high-dose chemotherapy with stem cell transplantation

  3. ECOG performance status score of 0,1,or2 -

Exclusion Criteria
  1. The current diagnosis of smoldering multiple myeloma, monoclonal gammopathy of undetermined significance of disease, Waldenstr o m macroglobulinemia.
  2. during the first 5 years of the study, there were no other malignancies, including basal cell carcinoma or in situ cervical cancer.
  3. according to the National Cancer Institute general toxicity criteria (NCI CTC), subjects had peripheral neuropathy of grade 2 or above:
  4. were enrolled within 6 months before had a myocardial infarction, or New York Heart Association (NYHA) III or IV heart failure ,uncontrolled angina, uncontrolled severe ventricular arrhythmias or ECG evidence of acute ischemia or conduction system abnormalities and activity the clinical significance of pericardial disease, or cardiac amyloidosis -

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
A:Allogeneic Stem Cell Transplant GroupAllogeneic Hematopoietic Stem Cell TransplantationFludarabine+Melphalan followed by Allogeneic SCT.
A:Allogeneic Stem Cell Transplant GroupFludarabine InjectionFludarabine+Melphalan followed by Allogeneic SCT.
A:Allogeneic Stem Cell Transplant GroupPI and dexamethasone as maintenance therapyFludarabine+Melphalan followed by Allogeneic SCT.
A:Allogeneic Stem Cell Transplant GroupMelphalan Given IVFludarabine+Melphalan followed by Allogeneic SCT.
B:Autologous Stem Cell TransplantAutologous Hematopoietic Stem Cell Transplantation x 1 or x 2Melphalan followed by Autologous SCT.
B:Autologous Stem Cell TransplantPI and dexamethasone as maintenance therapyMelphalan followed by Autologous SCT.
C:Non-TransplantPI and dexamethasone as maintenance therapyConsolidated Chemotherapy for Patients Unable to Receive Transplantation
C:Non-TransplantPI+IMids+Dexamethasone as Consolidated ChemotherapyConsolidated Chemotherapy for Patients Unable to Receive Transplantation
B:Autologous Stem Cell TransplantMelphalan Given IVMelphalan followed by Autologous SCT.
Primary Outcome Measures
NameTimeMethod
progression free survival(PFS)1 Year post-autograft

PFS is defined as the duration from the data of registration to either progressive disease or death, whichever comes first.

Secondary Outcome Measures
NameTimeMethod
Non-relapse Mortality (NRM)1 year post-allograft

Number of patients with non-relapse mortalities

overall survival(OS)1 Year post-autograft

OS is defined as the duration from the data of registration to death.If the subject is alive, the data will be censored as being alive; the vital status is unknown as last known.

Number of Patients Who Had Infections1 Year post-autograft

Number of patients who had infections

overall response(ORR)1 Year post-autograft

ORR is defined as the proportion of subjects who achieve PR to better rate, according to the IMWG criteria

Number of Patients With Grade II-IV Acute Graft-versus-Host-Disease and/or Chronic Extensive Graft-versus-Host-Disease1 year post-allograft

aGVHD The diagnosis of aGVHD is identified through various stages and grading of the disease related to Skin (Rash), Gut (Diarrhea, Nausea/vomiting and/or anorexia) and the liver (Bilirubin) assessed by severity and grading scale outlined in the section Grafts vs Hosts by Sullivan (1999).

GVHD Grades Grade I: 1-2 Skin Rash; No gut or liver involvement Grade II: Stage 1-3 Skin rash; Stage 1 gut and/or stage 1 liver involvement Grade III: Stage 2-4 gut involvement and/or stage 2-4 liver involvement with or without rash Grade IV: Pattern and severity of GVHD similar to grade 3 with extreme constitutional symptoms or death cGVHD The diagnosis of cGVHD requires at least one manifestation that is distinctive for chronic GVHD as opposed to acute GVHD. In all cases, infection and others causes must be ruled out in the differential diagnosis of chronic GVHD.

Trial Locations

Locations (1)

Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences

🇨🇳

Tianjin, Tianjin, China

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