NCT00301834 · ADVERSE EVENTS

Adverse events

Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders · Source last updated 2017-09-28

Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.

Adverse events occurred during the study; they are not necessarily caused by treatment. Serious events are not interchangeable with severe/grade 3–4 events. These safety populations may differ from efficacy populations.

Collection time frame: Not reported
Other-event reporting threshold: 5%

Participants affected / participants at risk, as reported
Safety group	Serious events	Other events	Deaths
Single Arm - Transplant Pre-conditioning Per Study Protocol All eligible patients received pre-transplant conditioning with Alemtuzumab, fludarabine and targeted busulfan followed by allogeneic transplant. The initial dose of busulfan was determined by performing PK (pharmacokinetics) analysis on a test dose of busulfan on the day prior to initiating conditioning. Based on the PK results a starting dose of busulfan was determined. A second PK study was done after the starting dose to ensure that the targeted dose was achieved. If it wasn't achieved, then a dose modification was made and a third PK study done.	7 / 35	33 / 35	Not reported / Not reported

Group identifiers: EG000: Single Arm - Transplant Pre-conditioning Per Study Protocol

Serious adverse events

Event terms and counts are source-reported; expand to read more. A reporting threshold may omit less frequent events.

assessment Type
SYSTEMATIC_ASSESSMENT
notes
acute Graft-Versus-Host Disease
organ System
General disorders
stats
group Id
EG000
num Affected
1
num At Risk
35
num Events
1
term
aGVHD (acute Graft-Versus-Host Disease)
assessment Type
NON_SYSTEMATIC_ASSESSMENT
notes
After 4th unrelated donor transplant (off study), patient developed respiratory distress that was associated with a CMV infection. Respiratory distress worsened in spite of maximal therapy. The parents elected to withdraw therapy and he died.
organ System
Respiratory, thoracic and mediastinal disorders
stats
group Id
EG000
num Affected
1
num At Risk
35
num Events
1
term
Respiratory distress associated with Cytomegalovirus infection
assessment Type
NON_SYSTEMATIC_ASSESSMENT
notes
Child presented with fever and pancytopenia, splenomegaly and was admitted for possible recurrent disease.
organ System
Blood and lymphatic system disorders
stats
group Id
EG000
num Affected
1
num At Risk
35
num Events
1
term
Pancytopenia and splenomegaly
assessment Type
NON_SYSTEMATIC_ASSESSMENT
notes
Worsening respiratory status despite maximal support, at parent's request, patient was removed from mechanical ventilation, and the patient died quickly and peacefully.
organ System
Respiratory, thoracic and mediastinal disorders
stats
group Id
EG000
num Affected
1
num At Risk
35
num Events
1
term
Repiratory failure following ideopathic pneumonia syndrome
assessment Type
NON_SYSTEMATIC_ASSESSMENT
notes
Sudden onset of thrombocytopenia of unknown etiology.
organ System
Blood and lymphatic system disorders
stats
group Id
EG000
num Affected
1
num At Risk
35
num Events
1
term
Thrombocytopenia
assessment Type
NON_SYSTEMATIC_ASSESSMENT
notes
Patient admitted with cough and fever and diagnosed with Aspergillus pneumonia.
organ System
Infections and infestations
stats
group Id
EG000
num Affected
1
num At Risk
35
num Events
1
term
Aspergillus pneumonia
assessment Type
NON_SYSTEMATIC_ASSESSMENT
notes
Patient developed renal failure and is required dialysis
organ System
Renal and urinary disorders
stats
group Id
EG000
num Affected
1
num At Risk
35
num Events
1
term
Renal Failure
assessment Type
SYSTEMATIC_ASSESSMENT
notes
initially, cells recovered normally with neutrophil count \>500 by day +12 post-transplant. By day +20 they began a slow decline and by day +40 the neutrophil count was \<500 with 99% host CD3+ (cluster of differentiation 3-positive) cells.
organ System
Blood and lymphatic system disorders
stats
group Id
EG000
num Affected
1
num At Risk
35
num Events
1
term
Graft rejection/failure
assessment Type
NON_SYSTEMATIC_ASSESSMENT
organ System
Infections and infestations
stats
group Id
EG000
num Affected
1
num At Risk
35
num Events
1
term
Viral Encephalitis
assessment Type
NON_SYSTEMATIC_ASSESSMENT
notes
Child died from a massive intracranial bleed, probably due to Evan's syndrome and myeloablation; possible viral encephalitis possibly increased risk of intracranial hemorrhage.
organ System
Vascular disorders
stats
group Id
EG000
num Affected
1
num At Risk
35
num Events
1
term
Massive intracranial bleed

Other reported adverse events

Event terms and counts are source-reported; expand to read more. A reporting threshold may omit less frequent events.

assessment Type
SYSTEMATIC_ASSESSMENT
notes
\< Grade III - 17 participants; Grade III - 15 participants; Grade IV - 1 participant
organ System
Gastrointestinal disorders
stats
group Id
EG000
num Affected
33
num At Risk
35
num Events
33
term
Mucositis (All grades)
assessment Type
SYSTEMATIC_ASSESSMENT
notes
aGVHD \< Grade IV
organ System
General disorders
stats
group Id
EG000
num Affected
5
num At Risk
35
num Events
5
term
aGVHD
assessment Type
SYSTEMATIC_ASSESSMENT
organ System
General disorders
stats
group Id
EG000
num Affected
2
num At Risk
35
num Events
2
term
cGVHD (Chronic graft-versus-host disease)
assessment Type
SYSTEMATIC_ASSESSMENT
organ System
General disorders
stats
group Id
EG000
num Affected
19
num At Risk
35
num Events
19
term
Infusion reaction to alemtuzumab

Complete source fields

event Groups

description
All eligible patients received pre-transplant conditioning with Alemtuzumab, fludarabine and targeted busulfan followed by allogeneic transplant. The initial dose of busulfan was determined by performing PK (pharmacokinetics) analysis on a test dose of busulfan on the day prior to initiating conditioning. Based on the PK results a starting dose of busulfan was determined. A second PK study was done after the starting dose to ensure that the targeted dose was achieved. If it wasn't achieved, then a dose modification was made and a third PK study done.
id
EG000
other Num Affected
33
other Num At Risk
35
serious Num Affected
7
serious Num At Risk
35
title
Single Arm - Transplant Pre-conditioning Per Study Protocol

frequency Threshold

other Events

assessment Type
SYSTEMATIC_ASSESSMENT
notes
\< Grade III - 17 participants; Grade III - 15 participants; Grade IV - 1 participant
organ System
Gastrointestinal disorders
stats
group Id
EG000
num Affected
33
num At Risk
35
num Events
33
term
Mucositis (All grades)
assessment Type
SYSTEMATIC_ASSESSMENT
notes
aGVHD \< Grade IV
organ System
General disorders
stats
group Id
EG000
num Affected
5
num At Risk
35
num Events
5
term
aGVHD
assessment Type
SYSTEMATIC_ASSESSMENT
organ System
General disorders
stats
group Id
EG000
num Affected
2
num At Risk
35
num Events
2
term
cGVHD (Chronic graft-versus-host disease)
assessment Type
SYSTEMATIC_ASSESSMENT
organ System
General disorders
stats
group Id
EG000
num Affected
19
num At Risk
35
num Events
19
term
Infusion reaction to alemtuzumab

serious Events

assessment Type
SYSTEMATIC_ASSESSMENT
notes
acute Graft-Versus-Host Disease
organ System
General disorders
stats
group Id
EG000
num Affected
1
num At Risk
35
num Events
1
term
aGVHD (acute Graft-Versus-Host Disease)
assessment Type
NON_SYSTEMATIC_ASSESSMENT
notes
After 4th unrelated donor transplant (off study), patient developed respiratory distress that was associated with a CMV infection. Respiratory distress worsened in spite of maximal therapy. The parents elected to withdraw therapy and he died.
organ System
Respiratory, thoracic and mediastinal disorders
stats
group Id
EG000
num Affected
1
num At Risk
35
num Events
1
term
Respiratory distress associated with Cytomegalovirus infection
assessment Type
NON_SYSTEMATIC_ASSESSMENT
notes
Child presented with fever and pancytopenia, splenomegaly and was admitted for possible recurrent disease.
organ System
Blood and lymphatic system disorders
stats
group Id
EG000
num Affected
1
num At Risk
35
num Events
1
term
Pancytopenia and splenomegaly
assessment Type
NON_SYSTEMATIC_ASSESSMENT
notes
Worsening respiratory status despite maximal support, at parent's request, patient was removed from mechanical ventilation, and the patient died quickly and peacefully.
organ System
Respiratory, thoracic and mediastinal disorders
stats
group Id
EG000
num Affected
1
num At Risk
35
num Events
1
term
Repiratory failure following ideopathic pneumonia syndrome
assessment Type
NON_SYSTEMATIC_ASSESSMENT
notes
Sudden onset of thrombocytopenia of unknown etiology.
organ System
Blood and lymphatic system disorders
stats
group Id
EG000
num Affected
1
num At Risk
35
num Events
1
term
Thrombocytopenia
assessment Type
NON_SYSTEMATIC_ASSESSMENT
notes
Patient admitted with cough and fever and diagnosed with Aspergillus pneumonia.
organ System
Infections and infestations
stats
group Id
EG000
num Affected
1
num At Risk
35
num Events
1
term
Aspergillus pneumonia
assessment Type
NON_SYSTEMATIC_ASSESSMENT
notes
Patient developed renal failure and is required dialysis
organ System
Renal and urinary disorders
stats
group Id
EG000
num Affected
1
num At Risk
35
num Events
1
term
Renal Failure
assessment Type
SYSTEMATIC_ASSESSMENT
notes
initially, cells recovered normally with neutrophil count \>500 by day +12 post-transplant. By day +20 they began a slow decline and by day +40 the neutrophil count was \<500 with 99% host CD3+ (cluster of differentiation 3-positive) cells.
organ System
Blood and lymphatic system disorders
stats
group Id
EG000
num Affected
1
num At Risk
35
num Events
1
term
Graft rejection/failure
assessment Type
NON_SYSTEMATIC_ASSESSMENT
organ System
Infections and infestations
stats
group Id
EG000
num Affected
1
num At Risk
35
num Events
1
term
Viral Encephalitis
assessment Type
NON_SYSTEMATIC_ASSESSMENT
notes
Child died from a massive intracranial bleed, probably due to Evan's syndrome and myeloablation; possible viral encephalitis possibly increased risk of intracranial hemorrhage.
organ System
Vascular disorders
stats
group Id
EG000
num Affected
1
num At Risk
35
num Events
1
term
Massive intracranial bleed

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