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Specialized Blood Cell Transplants for Cancers of the Blood and Bone Marrow
Phase 2 trial testing T-cell replete PBPC allograft in Myeloproliferative Disorders in 202 participants. Completed in 18 June 2020.
| Lead sponsor | National Heart, Lung, and Blood Institute (NHLBI) |
|---|---|
| Phase | Phase 2 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | non randomized |
| Design | parallel |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 202 |
| Start date | 15 April 1999 |
| Primary completion | 14 December 2018 |
| Estimated completion | 18 June 2020 |
| Sites | 1 location across United States |
National Heart, Lung, and Blood Institute (NHLBI)
Adults 2 to 80, any sex, with Myeloproliferative Disorders or Acute Myelogenous Leukemia. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants who experienced transplant related mortality by Day 200
| Group | Value | 95% CI |
|---|---|---|
| Group A: Stem Cell Transplant in High Risk for Transplant Related Complications and Mortality | 5 | |
| Group B: Stem Cell Transplant in Debilitating Hematologic Diseases | 4 |
Number of participants with complete donor myeloid chimerism. Myeloid (CD34+) and T-cell (CD3+) chimerisms were determined by PCR analysis of short tandem repeats (STR). Complete donor chimerism is defined as \>95% donor-derived cells in the peripheral blood in a specific lineage.
| Group | Value | 95% CI |
|---|---|---|
| Group A: Stem Cell Transplant in High Risk for Transplant Related Complications and Mortality | 29 | |
| Group B: Stem Cell Transplant in Debilitating Hematologic Diseases | 55 |
| Group | Value | 95% CI |
|---|---|---|
| Group A: Stem Cell Transplant in High Risk for Transplant Related Complications and Mortality | 30 | |
| Group B: Stem Cell Transplant in Debilitating Hematologic Diseases | 56 |
Median days to neutrophil recovery. Neutrophil recovery is defined as the first day of two consecutive days in which the ANC was 500 K/ml or greater unsupported by growth factors or granulocyte transfusion.
| Group | Value | 95% CI |
|---|---|---|
| Group A: Stem Cell Transplant in High Risk for Transplant Related Complications and Mortality | 14 | 7 – 22 |
| Group B: Stem Cell Transplant in Debilitating Hematologic Diseases | 15 | 6 – 24 |
Number of participants who experienced acute GVHD grades II-IV Acute-GVHD was graded and staged prospectively using criteria from the 1994 Consensus Conference on Acute-GVHD Grading. Grades are defined as: Grade II: Skin = rash on 25-50 percent body surface area; Liver = Total Bilirubin 3.1-6.0 mg/dL; Lower GI = Diarrhea 1001-1500 mL/day. Grade III: Skin = Rash on \>50% of body surface; Liver = Total Bilirubin 6.1 - 15.0 mg/dL; Lower GI = Diarrhea \> 1500 mL/day. Grade IV: Skin = Generalized erythroderma plus bullous formation; Liver = Total Bilirubin \>15 mg/dL; Lower GI = Severe abdomin
| Group | Value | 95% CI |
|---|---|---|
| Group A: Stem Cell Transplant in High Risk for Transplant Related Complications and Mortality | 6 | |
| Group B: Stem Cell Transplant in Debilitating Hematologic Diseases | 16 |
| Group | Value | 95% CI |
|---|---|---|
| Group A: Stem Cell Transplant in High Risk for Transplant Related Complications and Mortality | 14 | |
| Group B: Stem Cell Transplant in Debilitating Hematologic Diseases | 18 |
Number of participant who experienced chronic graft versus host disease (GVHD) following stem cell transplant The diagnosis of clinical features of chronic-GVHD was determined prospectively and classified retrospectively into limited or extensive based on the Revised Seattle Classification. Chronic GvHD severity categorized as "limited" is defined as: localized skin lesions with or without limited hepatic involvement and "extensive" is defined as: generalized skin involvement, major hepatic complications, or involvement of any other organ.
| Group | Value | 95% CI |
|---|---|---|
| Group A: Stem Cell Transplant in High Risk for Transplant Related Complications and Mortality | 12 | |
| Group B: Stem Cell Transplant in Debilitating Hematologic Diseases | 13 |
| Group | Value | 95% CI |
|---|---|---|
| Group A: Stem Cell Transplant in High Risk for Transplant Related Complications and Mortality | 19 | |
| Group B: Stem Cell Transplant in Debilitating Hematologic Diseases | 27 |
Number of participants overall survival. Overall survival is defined as number participants alive following stem cell transplant
| Group | Value | 95% CI |
|---|---|---|
| Group A: Stem Cell Transplant in High Risk for Transplant Related Complications and Mortality | 17 | |
| Group B: Stem Cell Transplant in Debilitating Hematologic Diseases | 48 |
Number of participants that remained Disease-free survival following stem cell transplant. Disease-free survival is defined as survival free of disease relapse or disease progression following stem cell transplant.
| Group | Value | 95% CI |
|---|---|---|
| Group A: Stem Cell Transplant in High Risk for Transplant Related Complications and Mortality | 26 | |
| Group B: Stem Cell Transplant in Debilitating Hematologic Diseases | 56 |
Time frame: 5 years. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Group A: Stem Cell Transpl… | Group B: Stem Cell Transpl… | Donor |
|---|---|---|---|---|
| Acute Gastrointestional Graft Verses Host Disease | Immune system disorders | — | — | — |
| Bacteremia | Infections and infestations | — | — | — |
| Pneumonia | Infections and infestations | — | — | — |
| Pain | General disorders | — | — | — |
| Sinusitis | Infections and infestations | — | — | — |
| CMV Reactivation | Infections and infestations | — | — | — |
| Fever | General disorders | — | — | — |
| Hyperglycemia | Investigations | — | — | — |
| Respiratory syncytial virus | Infections and infestations | — | — | — |
| Diarrhea | Gastrointestinal disorders | — | — | — |
| Disease progression | General disorders | — | — | — |
| Herpes zoster reactivation | Infections and infestations | — | — | — |
| Influenza | Infections and infestations | — | — | — |
| PRES | Nervous system disorders | — | — | — |
| Seizure | Nervous system disorders | — | — | — |
| Sepsis | Infections and infestations | — | — | — |
| Upper Respirtory Infection | Infections and infestations | — | — | — |
| Acute Graft verses Host Disease of Skin | Immune system disorders | — | — | — |
| Acute Myocardial Infarction | Cardiac disorders | — | — | — |
| Aspergillosis | Infections and infestations | — | — | — |
| Catheter Related Thrombosus | Vascular disorders | — | — | — |
| Cellulitis | Infections and infestations | — | — | — |
| Cholecystitis | Hepatobiliary disorders | — | — | — |
| Chronic Graft Verses Host Disease of Liver | Immune system disorders | — | — | — |
| Clostridioides difficile | Infections and infestations | — | — | — |
Most-reported serious reactions: Acute Gastrointestional Graft Verses Host Disease, Bacteremia, Pneumonia, Pain, Sinusitis, CMV Reactivation, Fever, Hyperglycemia.
Data from ClinicalTrials.gov NCT00003838 adverse events section.
The are a variety of cancerous diseases of the blood and bone marrow that can be potentially cured by bone marrow transplantation (BMT). Diseases like leukemia, lymphoma, and multiple myeloma are among the conditions that can be treated with BMT. Some patients with these diseases can be treated with medical chemotherapy alone. However, patients who relapse following chemotherapy are usually not curable with additional chemotherapy treatments. The only option known to provide a potential cure if this occurs is BMT. Allogenic transplants are cells collected from relatives of the patient. The transplant requires additional high intensity chemotherapy and radiation in order to destroy cancerous cells. In the process, many normal bone marrow cells are also destroyed. This is the reason for transplanting stem cells. The stem cells help to build new functioning bone marrow, red cells, white cells, and platelets. In addition, the immune cells from the donor are implanted into the recipient s body and help to fight off infection and kill remaining cancerous cells. Unfortunately, the powerful doses of chemotherapy and radiation therapy associated with allogenic BMT have toxic side effects and often make BMTs too dangerous to attempt in many patients. In order to reduce the complications of BMT, and make it a safer available option for patients with cancers of the blood and bone marrow, researchers have developed a new approach to the BMT. In this study researchers plan to use stem cells collected from the blood stream of patient s relatives rather than from the bone marrow (blood progenitor/stem cell transplant). In addition, researchers plan to use low doses of chemotherapy and no radiation therapy to reduce side effects. The majority of the cancer killing effect will be the responsibility of the stem cell transplant rather than the chemotherapy.
5 peer-reviewed publications reference this trial (live from Europe PMC):
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