Three Immunosuppressive Treatment Regimens for Severe Aplastic Anemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.

ClinicalTrials.gov Identifier: NCT00260689

Recruitment Status : Completed

First Posted : December 1, 2005

Results First Posted : June 8, 2017

Last Update Posted : June 8, 2017

Sponsor:

Information provided by (Responsible Party):

National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )

Study Type	Interventional
Study Design	Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment
Conditions	Immunosuppresion Thrombocytopenia Pancytopenia Neutropenia
Interventions	Biological: Anti-thymocyte globulin (rabbit) Biological: Anti-thymocyte globulin (horse) Drug: Cyclosporine Drug: Alemtuzumab
Enrollment	136

Participant Flow

Recruitment Details
Pre-assignment Details


Arm/Group Title	Horse ATG/CsA Taper	Rabbit ATG/CsA	Alemtuzumab
Arm/Group Description	Horse Anti-thymocyte Globulin (h-AT...	Rabbit Anti-thymocyte Globulin (r-A...	Alemtuzumab (Campath) administered ...
Arm/Group Description	Horse Anti-thymocyte Globulin (h-ATG) + 6 months Cyclosporine (CsA) followed by an 18 month CsA taper	Rabbit Anti-thymocyte Globulin (r-ATG) + 6 months Cyclosporine (CsA)	Alemtuzumab (Campath) administered for 10 days
Period Title: Overall Study
Started	60	60	16
Completed	19	11	1
Not Completed	41	49	15

Baseline Characteristics

Arm/Group Title		Horse ATG/CsA Taper	Rabbit ATG/CsA	Alemtuzumab	Total
Arm/Group Description		Horse Anti-thymocyte Globulin (h-AT...	Rabbit Anti-thymocyte Globulin (r-A...	Alemtuzumab (Campath) administered ...	Total of all reporting groups
Arm/Group Description		Horse Anti-thymocyte Globulin (h-ATG) + 6 months Cyclosporine (CsA) followed by an 18 month CsA taper	Rabbit Anti-thymocyte Globulin (r-ATG) + 6 months Cyclosporine (CsA)	Alemtuzumab (Campath) administered for 10 days	Total of all reporting groups
Overall Number of Baseline Participants		60	60	16	136
Baseline Analysis Population Description		[Not Specified]
Baseline Analysis Population Description		[Not Specified]
Age, Categorical Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	60 participants	60 participants	16 participants	136 participants
	<=18 years	14 23.3%	21 35.0%	4 25.0%	39 28.7%
	Between 18 and 65 years	38 63.3%	35 58.3%	8 50.0%	81 59.6%
	>=65 years	8 13.3%	4 6.7%	4 25.0%	16 11.8%
Sex: Female, Male Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	60 participants	60 participants	16 participants	136 participants
	Female	27 45.0%	23 38.3%	4 25.0%	54 39.7%
	Male	33 55.0%	37 61.7%	12 75.0%	82 60.3%
Ethnicity (NIH/OMB) Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	60 participants	60 participants	16 participants	136 participants
	Hispanic or Latino	9 15.0%	13 21.7%	7 43.8%	29 21.3%
	Not Hispanic or Latino	50 83.3%	44 73.3%	9 56.3%	103 75.7%
	Unknown or Not Reported	1 1.7%	3 5.0%	0 0.0%	4 2.9%
Race (NIH/OMB) Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	60 participants	60 participants	16 participants	136 participants
	American Indian or Alaska Native	0 0.0%	0 0.0%	0 0.0%	0 0.0%
	Asian	5 8.3%	2 3.3%	1 6.3%	8 5.9%
	Native Hawaiian or Other Pacific Islander	0 0.0%	0 0.0%	0 0.0%	0 0.0%
	Black or African American	15 25.0%	20 33.3%	1 6.3%	36 26.5%
	White	29 48.3%	24 40.0%	7 43.8%	60 44.1%
	More than one race	2 3.3%	0 0.0%	0 0.0%	2 1.5%
	Unknown or Not Reported	9 15.0%	14 23.3%	7 43.8%	30 22.1%

Outcome Measures

1.Primary Outcome


Title	Hematologic Response
Description	Hematologic response is ...
Description	Hematologic response is defined as subjects having blood counts no longer meeting the standard ("Camitta") criteria for severe pancytopenia in Severe Aplastic Anemia, equivalent to 2 of the following values obtained on 2 serial blood count measurements at least one week apart at landmark time points (3, 6 and 12 months) Absolute neutrophil count > 500/ μL Platelet count > 20,000/ μL Reticulocyte count > 60,000/ μL Improvement in counts that are dependent upon exogenously administered growth factors or transfusion will not be considered as fulfilling response criteria.
Time Frame	3 months

Outcome Measure Data

Analysis Population Description

[Not Specified]


Arm/Group Title	Horse ATG/CsA Taper	Rabbit ATG/CsA	Alemtuzumab
Arm/Group Description:	Horse Anti-thymocyte Globulin (h-AT...	Rabbit Anti-thymocyte Globulin (r-A...	Alemtuzumab (Campath) administered ...
Arm/Group Description:	Horse Anti-thymocyte Globulin (h-ATG) + 6 months Cyclosporine (CsA) followed by an 18 month CsA taper	Rabbit Anti-thymocyte Globulin (r-ATG) + 6 months Cyclosporine (CsA)	Alemtuzumab (Campath) administered for 10 days
Overall Number of Participants Analyzed	60	60	16
Measure Type: Count of Participants Unit of Measure: Participants
	38 63.3%	20 33.3%	3 18.8%

2.Primary Outcome


Title	Hematologic Response
Description	Hematologic response is ...
Description	Hematologic response is defined as subjects having blood counts no longer meeting the standard ("Camitta") criteria for severe pancytopenia in Severe Aplastic Anemia, equivalent to 2 of the following values obtained on 2 serial blood count measurements at least one week apart at landmark time points (3, 6 and 12 months) Absolute neutrophil count > 500/ μL Platelet count > 20,000/ μL Reticulocyte count > 60,000/ μL Improvement in counts that are dependent upon exogenously administered growth factors or transfusion will not be considered as fulfilling response criteria.
Time Frame	6 months

Outcome Measure Data

Analysis Population Description

[Not Specified]


Arm/Group Title	Horse ATG/CsA Taper	Rabbit ATG/CsA	Alemtuzumab
Arm/Group Description:	Horse Anti-thymocyte Globulin (h-AT...	Rabbit Anti-thymocyte Globulin (r-A...	Alemtuzumab (Campath) administered ...
Arm/Group Description:	Horse Anti-thymocyte Globulin (h-ATG) + 6 months Cyclosporine (CsA) followed by an 18 month CsA taper	Rabbit Anti-thymocyte Globulin (r-ATG) + 6 months Cyclosporine (CsA)	Alemtuzumab (Campath) administered for 10 days
Overall Number of Participants Analyzed	60	60	16
Measure Type: Count of Participants Unit of Measure: Participants
	41 68.3%	22 36.7%	3 18.8%

3.Primary Outcome


Title	Hematologic Response
Description	Hematologic response is ...
Description	Hematologic response is defined as subjects having blood counts no longer meeting the standard ("Camitta") criteria for severe pancytopenia in Severe Aplastic Anemia, equivalent to 2 of the following values obtained on 2 serial blood count measurements at least one week apart at landmark time points (3, 6 and 12 months) Absolute neutrophil count > 500/ μL Platelet count > 20,000/ μL Reticulocyte count > 60,000/ μL Improvement in counts that are dependent upon exogenously administered growth factors or transfusion will not be considered as fulfilling response criteria.
Time Frame	12 months

Outcome Measure Data

Analysis Population Description

[Not Specified]


Arm/Group Title	Horse ATG/CsA Taper	Rabbit ATG/CsA	Alemtuzumab
Arm/Group Description:	Horse Anti-thymocyte Globulin (h-AT...	Rabbit Anti-thymocyte Globulin (r-A...	Alemtuzumab (Campath) administered ...
Arm/Group Description:	Horse Anti-thymocyte Globulin (h-ATG) + 6 months Cyclosporine (CsA) followed by an 18 month CsA taper	Rabbit Anti-thymocyte Globulin (r-ATG) + 6 months Cyclosporine (CsA)	Alemtuzumab (Campath) administered for 10 days
Overall Number of Participants Analyzed	60	60	16
Measure Type: Count of Participants Unit of Measure: Participants
	40 66.7%	18 30.0%	2 12.5%

Adverse Events

Time Frame	5 years
Adverse Event Reporting Description	[Not Specified]

Arm/Group Title	Horse ATG/CsA Taper	Rabbit ATG/CsA	Alemtuzumab
Arm/Group Description	Horse Anti-thymocyte Globulin (h-AT...	Rabbit Anti-thymocyte Globulin (r-A...	Alemtuzumab (Campath) administered ...
Arm/Group Description	Horse Anti-thymocyte Globulin (h-ATG) + 6 months Cyclosporine (CsA) followed by an 18 month CsA taper	Rabbit Anti-thymocyte Globulin (r-ATG) + 6 months Cyclosporine (CsA)	Alemtuzumab (Campath) administered for 10 days
All-Cause Mortality
	Horse ATG/CsA Taper	Rabbit ATG/CsA	Alemtuzumab
	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)
Total	11/60 (18.33%)	18/60 (30.00%)	7/16 (43.75%)
Serious Adverse Events Serious Adverse Events
	Horse ATG/CsA Taper	Rabbit ATG/CsA	Alemtuzumab
	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)
Total	7/60 (11.67%)	11/60 (18.33%)	6/16 (37.50%)
Blood and lymphatic system disorders
CNS hemorrhage/bleeding ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Edema: limb ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Cardiac disorders
Cardiac troponin I ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
Hypertension ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Supraventricular arrhythmia ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Immune system disorders
Serum sickness ^†	2/60 (3.33%)	4/60 (6.67%)	0/16 (0.00%)
Serum sickness/Infection ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Infections and infestations
Infection (documented clinically or microbiologically with grade 3 or 4 neutropenia) ^†	3/60 (5.00%)	1/60 (1.67%)	5/16 (31.25%)
Infection without neutropenia ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Metabolism and nutrition disorders
Creatinine (grade 2) ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
Hypokalemia ^†	0/60 (0.00%)	0/60 (0.00%)	1/16 (6.25%)
Musculoskeletal and connective tissue disorders
Arthralgia (joint pain) ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
Nervous system disorders
Seizures ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
† Indicates events were collected by systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	Horse ATG/CsA Taper	Rabbit ATG/CsA	Alemtuzumab
	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)
Total	36/60 (60.00%)	39/60 (65.00%)	15/16 (93.75%)
Blood and lymphatic system disorders
edema ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
edema - mild lower extremity ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
gingival hypertrophy ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Hemolysis ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
lower extremity edema ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
lower extremity swelling ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
slight edema pitting ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
slight pitting edema ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
swelling (lower extremity) ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
swelling ankles ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
swelling feet and hands ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Cardiac disorders
bradycardia ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Edema, ankles ^†	2/60 (3.33%)	1/60 (1.67%)	0/16 (0.00%)
Edema, lower legs ^†	3/60 (5.00%)	1/60 (1.67%)	1/16 (6.25%)
fluid overload ^†	2/60 (3.33%)	1/60 (1.67%)	0/16 (0.00%)
h Blood pressure ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
h BP ^†	3/60 (5.00%)	1/60 (1.67%)	0/16 (0.00%)
high blood pressure ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
Hypertension ^†	8/60 (13.33%)	9/60 (15.00%)	3/16 (18.75%)
hypotension ^†	2/60 (3.33%)	3/60 (5.00%)	0/16 (0.00%)
i SBP ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
irregular pulse ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
S tachycardia ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
substernal pressure ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Supraventricular tachycardia ^†	0/60 (0.00%)	0/60 (0.00%)	1/16 (6.25%)
Tachycardia ^†	2/60 (3.33%)	1/60 (1.67%)	1/16 (6.25%)
Endocrine disorders
Hot flashes ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Hypothyroidism, subclinical ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Eye disorders
Photophobia ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Gastrointestinal disorders
Abdominal discomfort ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
abdominal distension ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
abdominal gas ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
abdominal pain ^†	2/60 (3.33%)	0/60 (0.00%)	0/16 (0.00%)
abdominal pain with loose stools ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
bleeding, swollen gums ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
bloated ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
decreased ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
dehydration ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
diarrhea ^†	5/60 (8.33%)	8/60 (13.33%)	0/16 (0.00%)
Gingival hyperplasia ^†	2/60 (3.33%)	4/60 (6.67%)	2/16 (12.50%)
gingivial hyperplasia ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
gingivial hyperplasia - mild ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
gingivitis ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
gum hyperplasia ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
gum hypertrophy ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Heartburn ^†	3/60 (5.00%)	1/60 (1.67%)	1/16 (6.25%)
i K+ ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Indigestion ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
loose stool ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
Nausea ^†	3/60 (5.00%)	4/60 (6.67%)	0/16 (0.00%)
nausea + vomiting ^†	2/60 (3.33%)	1/60 (1.67%)	0/16 (0.00%)
poor appetite ^†	1/60 (1.67%)	1/60 (1.67%)	0/16 (0.00%)
vomiting ^†	2/60 (3.33%)	3/60 (5.00%)	0/16 (0.00%)
General disorders
Abdominal ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
abdominal pain ^†	1/60 (1.67%)	2/60 (3.33%)	0/16 (0.00%)
Abdominal, cramps ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
body aches ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
chest discomfort ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
chest pain ^†	0/60 (0.00%)	2/60 (3.33%)	0/16 (0.00%)
Chest pressure ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
chest tightness ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
chills ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
chills/rigors ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
epigastric pain ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
fever ^†	6/60 (10.00%)	5/60 (8.33%)	0/16 (0.00%)
Headache ^†	10/60 (16.67%)	6/60 (10.00%)	1/16 (6.25%)
leg pain ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Localized, back ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Localized, feet (burning), knee ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Localized, low back ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
Localized, soles of feet, legs ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
Localized, Throat ^†	0/60 (0.00%)	0/60 (0.00%)	1/16 (6.25%)
Localized, throat, abdomen ^†	0/60 (0.00%)	0/60 (0.00%)	1/16 (6.25%)
Localized, Wrist, bilateral ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
low back pain ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
Myalgia (general) ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Myalgia, arthralgia ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Myalgia, back ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Myalgia, calves ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
neutropenic fever ^†	2/60 (3.33%)	3/60 (5.00%)	0/16 (0.00%)
retrosternal chest pain ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
rigor ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
rigors ^†	5/60 (8.33%)	2/60 (3.33%)	0/16 (0.00%)
stomach discomfort ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
tired ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
weight gain ^†	2/60 (3.33%)	2/60 (3.33%)	0/16 (0.00%)
weight increase ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
weight loss ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Hepatobiliary disorders
Elevated LFT's ^†	9/60 (15.00%)	9/60 (15.00%)	5/16 (31.25%)
Elevated t. bili ^†	5/60 (8.33%)	6/60 (10.00%)	0/16 (0.00%)
Immune system disorders
flu-like symptoms ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
joint Pain ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Labeled infusion related ^†	28/60 (46.67%)	31/60 (51.67%)	15/16 (93.75%)
Serum sickness ^†	2/60 (3.33%)	5/60 (8.33%)	0/16 (0.00%)
swelling ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
throat swelling ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Infections and infestations
C. diff colitis ^†	0/60 (0.00%)	3/60 (5.00%)	0/16 (0.00%)
C. diff colitis and Blastocystis hominis ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Gingivitis ^†	3/60 (5.00%)	1/60 (1.67%)	0/16 (0.00%)
Mouth sores ^†	3/60 (5.00%)	3/60 (5.00%)	1/16 (6.25%)
Mouth sores (neg. Cxs) ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Shingles ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
Trush ^†	1/60 (1.67%)	1/60 (1.67%)	1/16 (6.25%)
Upper Respiratory Tract ^†	0/60 (0.00%)	1/60 (1.67%)	1/16 (6.25%)
Metabolism and nutrition disorders
h ALT ^†	1/60 (1.67%)	1/60 (1.67%)	0/16 (0.00%)
h ALT/di bili ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
h AST ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
h creatinine ^†	3/60 (5.00%)	4/60 (6.67%)	0/16 (0.00%)
h creatinine -mild ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
h K ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
h LDH ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
h LFT ^†	2/60 (3.33%)	0/60 (0.00%)	0/16 (0.00%)
h LFT, creatinine ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
h LFTs ^†	1/60 (1.67%)	1/60 (1.67%)	0/16 (0.00%)
h PT, PTT ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Hyperglicemia ^†	2/60 (3.33%)	2/60 (3.33%)	1/16 (6.25%)
hyperglycemia ^†	1/60 (1.67%)	1/60 (1.67%)	0/16 (0.00%)
hyperkalemia ^†	2/60 (3.33%)	0/60 (0.00%)	0/16 (0.00%)
hypernatremia ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
hypertension ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
hypoglycemia ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
hypokalemia ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
hypomagnesia ^†	1/60 (1.67%)	1/60 (1.67%)	0/16 (0.00%)
i magnesium ^†	2/60 (3.33%)	2/60 (3.33%)	0/16 (0.00%)
LFT abnormal ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
low albumin ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
renal failure ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
Musculoskeletal and connective tissue disorders
Bursitis ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Cramps ^†	1/60 (1.67%)	1/60 (1.67%)	0/16 (0.00%)
Nervous system disorders
anxiety ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
Anxiety, irritability ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
dizziness ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Insomnia ^†	0/60 (0.00%)	2/60 (3.33%)	1/16 (6.25%)
numbness ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
Numbness fingers, wrists ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
numbness/tingling (bilateral lower extremities) ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
parastesias ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Perioral tingling ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
Peripheral neuropathy, CsA induced ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
tingling ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Tremor, hands ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
tremors ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Renal and urinary disorders
acute kidney injury ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
Dysuria ^†	0/60 (0.00%)	1/60 (1.67%)	1/16 (6.25%)
mild renal insufficiency ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
Respiratory, thoracic and mediastinal disorders
cough ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
crackles ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
crackles in lungs ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
crackles lungs ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
dry cough ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
short of breath ^†	1/60 (1.67%)	1/60 (1.67%)	0/16 (0.00%)
sob ^†	2/60 (3.33%)	1/60 (1.67%)	0/16 (0.00%)
Skin and subcutaneous tissue disorders
Acne ^†	1/60 (1.67%)	1/60 (1.67%)	0/16 (0.00%)
Body hair growth ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
Dandruff ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
Erythema, LE ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
erythematous rash ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
hair growth ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
increased hair growth ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
itchy palms ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
palm itching ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
palms slightly red ^†	0/60 (0.00%)	1/60 (1.67%)	0/16 (0.00%)
Rash ^†	2/60 (3.33%)	2/60 (3.33%)	0/16 (0.00%)
rash (arms) ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
rash - mild ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
rash face ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
Rash, face ^†	0/60 (0.00%)	0/60 (0.00%)	1/16 (6.25%)
rash, hives, itching ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
urticarial rash -mild ^†	1/60 (1.67%)	0/60 (0.00%)	0/16 (0.00%)
† Indicates events were collected by systematic assessment

Limitations and Caveats

Originally, there were 3 regimens: h-ATG/CsA ; r-ATG/CsA; alemtuzumab. Subjects not responding to r-ATG will cross over to alemtuzumab, and subjects failing alemtuzumab will cross over to r-ATG. Subjects not responding to h-ATG/CsA will go off study.

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

Results Point of Contact

Layout table for Results Point of Contact information

Name/Title:	Danielle Townsley MD
Organization:	NHLBI, NIH
Phone:	301-402-3477
EMail:	townsleydm@nhlbi.nih.gov

Publications:

Young NS, Barrett AJ. The treatment of severe acquired aplastic anemia. Blood. 1995 Jun 15;85(12):3367-77. Review.

Young NS, Maciejewski J. The pathophysiology of acquired aplastic anemia. N Engl J Med. 1997 May 8;336(19):1365-72. Review.

Zoumbos NC, Gascón P, Djeu JY, Trost SR, Young NS. Circulating activated suppressor T lymphocytes in aplastic anemia. N Engl J Med. 1985 Jan 31;312(5):257-65.

Feng X, Scheinberg P, Biancotto A, Rios O, Donaldson S, Wu C, Zheng H, Sato K, Townsley DM, McCoy JP, Young NS. In vivo effects of horse and rabbit antithymocyte globulin in patients with severe aplastic anemia. Haematologica. 2014 Sep;99(9):1433-40. doi: 10.3324/haematol.2014.106542. Epub 2014 Jun 6.

Scheinberg P, Townsley D, Dumitriu B, Scheinberg P, Weinstein B, Rios O, Wu CO, Young NS. Horse antithymocyte globulin as salvage therapy after rabbit antithymocyte globulin for severe aplastic anemia. Am J Hematol. 2014 May;89(5):467-9. doi: 10.1002/ajh.23669. Epub 2014 Mar 7.

Scheinberg P, Nunez O, Weinstein B, Scheinberg P, Wu CO, Young NS. Activity of alemtuzumab monotherapy in treatment-naive, relapsed, and refractory severe acquired aplastic anemia. Blood. 2012 Jan 12;119(2):345-54. doi: 10.1182/blood-2011-05-352328. Epub 2011 Nov 8.

Scheinberg P, Nunez O, Weinstein B, Scheinberg P, Biancotto A, Wu CO, Young NS. Horse versus rabbit antithymocyte globulin in acquired aplastic anemia. N Engl J Med. 2011 Aug 4;365(5):430-8. doi: 10.1056/NEJMoa1103975. Erratum in: N Engl J Med. 2018 Jun 13;:null.

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Giudice V, Wu Z, Kajigaya S, Fernandez Ibanez MDP, Rios O, Cheung F, Ito S, Young NS. Circulating S100A8 and S100A9 protein levels in plasma of patients with acquired aplastic anemia and myelodysplastic syndromes. Cytokine. 2019 Jan;113:462-465. doi: 10.1016/j.cyto.2018.06.025. Epub 2018 Jun 27.

Giudice V, Banaszak LG, Gutierrez-Rodrigues F, Kajigaya S, Panjwani R, Ibanez MDPF, Rios O, Bleck CK, Stempinski ES, Raffo DQ, Townsley DM, Young NS. Circulating exosomal microRNAs in acquired aplastic anemia and myelodysplastic syndromes. Haematologica. 2018 Jul;103(7):1150-1159. doi: 10.3324/haematol.2017.182824. Epub 2018 Apr 19.

Hosokawa K, Kajigaya S, Feng X, Desierto MJ, Fernandez Ibanez MD, Rios O, Weinstein B, Scheinberg P, Townsley DM, Young NS. A plasma microRNA signature as a biomarker for acquired aplastic anemia. Haematologica. 2017 Jan;102(1):69-78. doi: 10.3324/haematol.2016.151076. Epub 2016 Sep 22.

Layout table for additonal information

Responsible Party:	National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
ClinicalTrials.gov Identifier:	NCT00260689
Other Study ID Numbers:	060034 06-H-0034 ( Other Identifier: NIH )
First Submitted:	December 1, 2005
First Posted:	December 1, 2005
Results First Submitted:	May 16, 2017
Results First Posted:	June 8, 2017
Last Update Posted:	June 8, 2017

To Top