Trial Outcomes & Findings for Preservation of Ovarian Function After Hematopoietic Cell Transplant (NCT NCT01343368)

NCT ID: NCT01343368

Last Updated: 2017-12-05

Results Overview

Comparison of treatment arms; interventional versus observational. Ovarian failure rate is based on FSH measured at 180 days after HCT; to determine the effect of GnRH agonists on the incidence of ovarian failure (i.e. FSH \>40 IU/L) after transplant.

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 19 participants
• Primary outcome timeframe: Through Day 180 Post Transplant
• Results posted on: 2017-12-05

Participant Flow

Participant milestones

Measure	Interventional - Received Leuprolide Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-HCT and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days for patients who undergo myeloablative allogeneic or autologous hematopoietic cell transplantation. Leuprolide: Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-transplant (HCT) and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days hematopoietic cell transplant: Conventional bone marrow transplant regimen.	Observational Arm Women undergoing reduced intensity allogeneic HCT will be observed. Progestin contraceptives, Norethindrone acetate, and any other hormone methods can be used according to the prescription guidelines except for GnRH agonists to suppress menses. reduced intensity allogeneic HCT: A reduced-intensity conditioning transplant is a bone marrow or cord blood transplant (also called a BMT) that uses less intense treatment to prepare for transplant than a standard transplant does. While a standard transplant uses the pre-transplant treatment to destroy most of the disease cells, a reduced-intensity transplant relies on the donor's immune cells to fight disease.
Overall Study STARTED	9	10
Overall Study COMPLETED	7	10
Overall Study NOT COMPLETED	2	0

Reasons for withdrawal

Measure	Interventional - Received Leuprolide Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-HCT and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days for patients who undergo myeloablative allogeneic or autologous hematopoietic cell transplantation. Leuprolide: Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-transplant (HCT) and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days hematopoietic cell transplant: Conventional bone marrow transplant regimen.	Observational Arm Women undergoing reduced intensity allogeneic HCT will be observed. Progestin contraceptives, Norethindrone acetate, and any other hormone methods can be used according to the prescription guidelines except for GnRH agonists to suppress menses. reduced intensity allogeneic HCT: A reduced-intensity conditioning transplant is a bone marrow or cord blood transplant (also called a BMT) that uses less intense treatment to prepare for transplant than a standard transplant does. While a standard transplant uses the pre-transplant treatment to destroy most of the disease cells, a reduced-intensity transplant relies on the donor's immune cells to fight disease.
Overall Study Death	1	0
Overall Study Lost to Follow-up	1	0

Baseline Characteristics

Preservation of Ovarian Function After Hematopoietic Cell Transplant

Baseline characteristics by cohort

Measure	Interventional - Received Leuprolide n=7 Participants Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-HCT and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days for patients who undergo myeloablative allogeneic or autologous hematopoietic cell transplantation. Leuprolide: Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-transplant (HCT) and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days hematopoietic cell transplant: Conventional bone marrow transplant regimen.	Observational Arm n=10 Participants Women undergoing reduced intensity allogeneic HCT will be observed. Progestin contraceptives, Norethindrone acetate, and any other hormone methods can be used according to the prescription guidelines except for GnRH agonists to suppress menses. reduced intensity allogeneic HCT: A reduced-intensity conditioning transplant is a bone marrow or cord blood transplant (also called a BMT) that uses less intense treatment to prepare for transplant than a standard transplant does. While a standard transplant uses the pre-transplant treatment to destroy most of the disease cells, a reduced-intensity transplant relies on the donor's immune cells to fight disease.	Total n=17 Participants Total of all reporting groups
Age, Categorical <=18 years	2 Participants n=5 Participants	6 Participants n=7 Participants	8 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=5 Participants	4 Participants n=7 Participants	9 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants	10 Participants n=7 Participants	17 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Through Day 180 Post Transplant

Comparison of treatment arms; interventional versus observational. Ovarian failure rate is based on FSH measured at 180 days after HCT; to determine the effect of GnRH agonists on the incidence of ovarian failure (i.e. FSH >40 IU/L) after transplant.

Outcome measures

Measure	Interventional - Received Leuprolide n=7 Participants Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-HCT and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days for patients who undergo myeloablative allogeneic or autologous hematopoietic cell transplantation. Leuprolide: Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-transplant (HCT) and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days hematopoietic cell transplant: Conventional bone marrow transplant regimen.	Observational Arm n=10 Participants Women undergoing reduced intensity allogeneic HCT will be observed. Progestin contraceptives, Norethindrone acetate, and any other hormone methods can be used according to the prescription guidelines except for GnRH agonists to suppress menses. reduced intensity allogeneic HCT: A reduced-intensity conditioning transplant is a bone marrow or cord blood transplant (also called a BMT) that uses less intense treatment to prepare for transplant than a standard transplant does. While a standard transplant uses the pre-transplant treatment to destroy most of the disease cells, a reduced-intensity transplant relies on the donor's immune cells to fight disease.
Comparison of Number of Patients With Ovarian Failure	3 Participants	1 Participants

SECONDARY outcome

Timeframe: From Baseline Through Day 365

Population: Five patients on the Interventional arm and 10 patients on the Observational arm were lost to follow-up by Day 365.

Comparison of treatment arms; interventional versus observational. Count of patients who stopped menstrual bleeding; to determine how the effect of GnRH agonists are at suppressing menses during hematopoietic cell transplant

Outcome measures

Measure	Interventional - Received Leuprolide n=2 Participants Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-HCT and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days for patients who undergo myeloablative allogeneic or autologous hematopoietic cell transplantation. Leuprolide: Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-transplant (HCT) and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days hematopoietic cell transplant: Conventional bone marrow transplant regimen.	Observational Arm Women undergoing reduced intensity allogeneic HCT will be observed. Progestin contraceptives, Norethindrone acetate, and any other hormone methods can be used according to the prescription guidelines except for GnRH agonists to suppress menses. reduced intensity allogeneic HCT: A reduced-intensity conditioning transplant is a bone marrow or cord blood transplant (also called a BMT) that uses less intense treatment to prepare for transplant than a standard transplant does. While a standard transplant uses the pre-transplant treatment to destroy most of the disease cells, a reduced-intensity transplant relies on the donor's immune cells to fight disease.
Comparison of Number of Patients Who Stopped Menstrual Bleeding	2 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline

Population: Four patients on the Observational arm never had any follow-up FSH levels drawn and were removed from this analysis.

Comparison of treatment arms; interventional versus observational average FSH levels.

Outcome measures

Measure	Interventional - Received Leuprolide n=7 Participants Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-HCT and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days for patients who undergo myeloablative allogeneic or autologous hematopoietic cell transplantation. Leuprolide: Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-transplant (HCT) and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days hematopoietic cell transplant: Conventional bone marrow transplant regimen.	Observational Arm n=6 Participants Women undergoing reduced intensity allogeneic HCT will be observed. Progestin contraceptives, Norethindrone acetate, and any other hormone methods can be used according to the prescription guidelines except for GnRH agonists to suppress menses. reduced intensity allogeneic HCT: A reduced-intensity conditioning transplant is a bone marrow or cord blood transplant (also called a BMT) that uses less intense treatment to prepare for transplant than a standard transplant does. While a standard transplant uses the pre-transplant treatment to destroy most of the disease cells, a reduced-intensity transplant relies on the donor's immune cells to fight disease.
Comparison of Follicle Stimulating Hormone (FSH) Levels	9.3 IU/L Standard Deviation 8.0	4.4 IU/L Standard Deviation 1.7

SECONDARY outcome

Timeframe: Day 100

Population: Four patients on the Observational arm never had any follow-up FSH levels drawn and were removed from this analysis. One patient on the Interventional arm and 7 on the Observational arm were lost to follow-up.

Comparison of treatment arms; interventional versus observational average FSH levels.

Outcome measures

Measure	Interventional - Received Leuprolide n=6 Participants Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-HCT and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days for patients who undergo myeloablative allogeneic or autologous hematopoietic cell transplantation. Leuprolide: Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-transplant (HCT) and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days hematopoietic cell transplant: Conventional bone marrow transplant regimen.	Observational Arm n=3 Participants Women undergoing reduced intensity allogeneic HCT will be observed. Progestin contraceptives, Norethindrone acetate, and any other hormone methods can be used according to the prescription guidelines except for GnRH agonists to suppress menses. reduced intensity allogeneic HCT: A reduced-intensity conditioning transplant is a bone marrow or cord blood transplant (also called a BMT) that uses less intense treatment to prepare for transplant than a standard transplant does. While a standard transplant uses the pre-transplant treatment to destroy most of the disease cells, a reduced-intensity transplant relies on the donor's immune cells to fight disease.
Comparison of Follicle Stimulating Hormone (FSH) Levels	18.4 IU/L Standard Deviation 19.5	9.1 IU/L Standard Deviation 12.7

SECONDARY outcome

Timeframe: Day 180

Population: Four patients on the Observational arm never had any follow-up FSH levels drawn and were removed from this analysis. One patient on the Interventional arm and 7 on the Observational arm were lost to follow-up.

Comparison of treatment arms; interventional versus observational average FSH levels.

Outcome measures

Measure	Interventional - Received Leuprolide n=6 Participants Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-HCT and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days for patients who undergo myeloablative allogeneic or autologous hematopoietic cell transplantation. Leuprolide: Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-transplant (HCT) and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days hematopoietic cell transplant: Conventional bone marrow transplant regimen.	Observational Arm n=3 Participants Women undergoing reduced intensity allogeneic HCT will be observed. Progestin contraceptives, Norethindrone acetate, and any other hormone methods can be used according to the prescription guidelines except for GnRH agonists to suppress menses. reduced intensity allogeneic HCT: A reduced-intensity conditioning transplant is a bone marrow or cord blood transplant (also called a BMT) that uses less intense treatment to prepare for transplant than a standard transplant does. While a standard transplant uses the pre-transplant treatment to destroy most of the disease cells, a reduced-intensity transplant relies on the donor's immune cells to fight disease.
Comparison of Follicle Stimulating Hormone (FSH) Levels	47.0 IU/L Standard Deviation 40.1	4.7 IU/L Standard Deviation 4.7

SECONDARY outcome

Timeframe: 1 year

Population: Four patients on the Observational arm never had any follow-up FSH levels drawn and were removed from this analysis. Three patients on the Interventional arm and 8 patients on the Observational arm were lost to follow-up.

Comparison of treatment arms; interventional versus observational average FSH levels.

Outcome measures

Measure	Interventional - Received Leuprolide n=4 Participants Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-HCT and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days for patients who undergo myeloablative allogeneic or autologous hematopoietic cell transplantation. Leuprolide: Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-transplant (HCT) and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days hematopoietic cell transplant: Conventional bone marrow transplant regimen.	Observational Arm n=2 Participants Women undergoing reduced intensity allogeneic HCT will be observed. Progestin contraceptives, Norethindrone acetate, and any other hormone methods can be used according to the prescription guidelines except for GnRH agonists to suppress menses. reduced intensity allogeneic HCT: A reduced-intensity conditioning transplant is a bone marrow or cord blood transplant (also called a BMT) that uses less intense treatment to prepare for transplant than a standard transplant does. While a standard transplant uses the pre-transplant treatment to destroy most of the disease cells, a reduced-intensity transplant relies on the donor's immune cells to fight disease.
Comparison of Follicle Stimulating Hormone (FSH) Levels	61.5 IU/L Standard Deviation 97.6	48.8 IU/L Standard Deviation 84.5

SECONDARY outcome

Timeframe: 2 years

Population: Four patients on the Observational arm never had any follow-up FSH levels drawn and were removed from this analysis. All 7 patients on the Interventional arm and 8 patients on the Observational arm were lost to follow-up.

Comparison of treatment arms; interventional versus observational average FSH levels.

Outcome measures

Measure	Interventional - Received Leuprolide Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-HCT and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days for patients who undergo myeloablative allogeneic or autologous hematopoietic cell transplantation. Leuprolide: Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-transplant (HCT) and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days hematopoietic cell transplant: Conventional bone marrow transplant regimen.	Observational Arm n=2 Participants Women undergoing reduced intensity allogeneic HCT will be observed. Progestin contraceptives, Norethindrone acetate, and any other hormone methods can be used according to the prescription guidelines except for GnRH agonists to suppress menses. reduced intensity allogeneic HCT: A reduced-intensity conditioning transplant is a bone marrow or cord blood transplant (also called a BMT) that uses less intense treatment to prepare for transplant than a standard transplant does. While a standard transplant uses the pre-transplant treatment to destroy most of the disease cells, a reduced-intensity transplant relies on the donor's immune cells to fight disease.
Comparison of Follicle Stimulating Hormone (FSH) Levels	—	7.1 IU/L Standard Deviation 4.6

SECONDARY outcome

Timeframe: Day 365 Post Transplant

Population: Only 6 of the 10 patients that started on the Observational Arm were evaluable. The 2 patients were lost to follow-up.

Comparison of treatment arms; interventional versus observational. Count of patients who resumed menses after hematopoietic cell transplant

Outcome measures

Measure	Interventional - Received Leuprolide n=7 Participants Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-HCT and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days for patients who undergo myeloablative allogeneic or autologous hematopoietic cell transplantation. Leuprolide: Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-transplant (HCT) and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days hematopoietic cell transplant: Conventional bone marrow transplant regimen.	Observational Arm n=6 Participants Women undergoing reduced intensity allogeneic HCT will be observed. Progestin contraceptives, Norethindrone acetate, and any other hormone methods can be used according to the prescription guidelines except for GnRH agonists to suppress menses. reduced intensity allogeneic HCT: A reduced-intensity conditioning transplant is a bone marrow or cord blood transplant (also called a BMT) that uses less intense treatment to prepare for transplant than a standard transplant does. While a standard transplant uses the pre-transplant treatment to destroy most of the disease cells, a reduced-intensity transplant relies on the donor's immune cells to fight disease.
Comparison of Number of Patients Who Resumed Menstrual Cycles	2 Participants	4 Participants

SECONDARY outcome

Timeframe: Baseline

Population: Four patients on the Observational arm never had any follow-up LH levels drawn and were removed from this analysis.

Comparison of treatment arms; interventional versus observational average LH levels during study.

Outcome measures

Measure	Interventional - Received Leuprolide n=7 Participants Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-HCT and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days for patients who undergo myeloablative allogeneic or autologous hematopoietic cell transplantation. Leuprolide: Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-transplant (HCT) and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days hematopoietic cell transplant: Conventional bone marrow transplant regimen.	Observational Arm n=6 Participants Women undergoing reduced intensity allogeneic HCT will be observed. Progestin contraceptives, Norethindrone acetate, and any other hormone methods can be used according to the prescription guidelines except for GnRH agonists to suppress menses. reduced intensity allogeneic HCT: A reduced-intensity conditioning transplant is a bone marrow or cord blood transplant (also called a BMT) that uses less intense treatment to prepare for transplant than a standard transplant does. While a standard transplant uses the pre-transplant treatment to destroy most of the disease cells, a reduced-intensity transplant relies on the donor's immune cells to fight disease.
Comparison of Lutineizing Hormone (LH) Levels	19.8 IU/L Standard Deviation 30.2	6.8 IU/L Standard Deviation 7.6

SECONDARY outcome

Timeframe: Day 100

Population: Four patients on the Observational arm never had any follow-up LH levels drawn and were removed from this analysis. One patient on the Interventional arm and 7 on the Observational arm were lost to follow-up.

Comparison of treatment arms; interventional versus observational average LH levels during study.

Outcome measures

Measure	Interventional - Received Leuprolide n=6 Participants Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-HCT and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days for patients who undergo myeloablative allogeneic or autologous hematopoietic cell transplantation. Leuprolide: Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-transplant (HCT) and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days hematopoietic cell transplant: Conventional bone marrow transplant regimen.	Observational Arm n=3 Participants Women undergoing reduced intensity allogeneic HCT will be observed. Progestin contraceptives, Norethindrone acetate, and any other hormone methods can be used according to the prescription guidelines except for GnRH agonists to suppress menses. reduced intensity allogeneic HCT: A reduced-intensity conditioning transplant is a bone marrow or cord blood transplant (also called a BMT) that uses less intense treatment to prepare for transplant than a standard transplant does. While a standard transplant uses the pre-transplant treatment to destroy most of the disease cells, a reduced-intensity transplant relies on the donor's immune cells to fight disease.
Comparison of Luteinizing Hormone (LH) Levels	2.4 IU/L Standard Deviation 2.7	6.4 IU/L Standard Deviation 7.7

SECONDARY outcome

Timeframe: Day 180

Population: Four patients on the Observational arm never had any follow-up LH levels drawn and were removed from this analysis. One patient on the Interventional arm and 7 on the Observational arm were lost to follow-up.

Comparison of treatment arms; interventional versus observational average LH levels during study.

Outcome measures

Measure	Interventional - Received Leuprolide n=6 Participants Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-HCT and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days for patients who undergo myeloablative allogeneic or autologous hematopoietic cell transplantation. Leuprolide: Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-transplant (HCT) and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days hematopoietic cell transplant: Conventional bone marrow transplant regimen.	Observational Arm n=3 Participants Women undergoing reduced intensity allogeneic HCT will be observed. Progestin contraceptives, Norethindrone acetate, and any other hormone methods can be used according to the prescription guidelines except for GnRH agonists to suppress menses. reduced intensity allogeneic HCT: A reduced-intensity conditioning transplant is a bone marrow or cord blood transplant (also called a BMT) that uses less intense treatment to prepare for transplant than a standard transplant does. While a standard transplant uses the pre-transplant treatment to destroy most of the disease cells, a reduced-intensity transplant relies on the donor's immune cells to fight disease.
Comparison of Luteinizing Hormone (LH) Levels	31.1 IU/L Standard Deviation 32.0	4.3 IU/L Standard Deviation 3.0

SECONDARY outcome

Timeframe: 1 year

Population: Four patients on the Observational arm never had any follow-up LH levels drawn and were removed from this analysis. Three patients on the Interventional arm and 8 patients on the Observational arm were lost to follow-up.

Comparison of treatment arms; interventional versus observational average LH levels during study.

Outcome measures

Measure	Interventional - Received Leuprolide n=4 Participants Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-HCT and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days for patients who undergo myeloablative allogeneic or autologous hematopoietic cell transplantation. Leuprolide: Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-transplant (HCT) and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days hematopoietic cell transplant: Conventional bone marrow transplant regimen.	Observational Arm n=2 Participants Women undergoing reduced intensity allogeneic HCT will be observed. Progestin contraceptives, Norethindrone acetate, and any other hormone methods can be used according to the prescription guidelines except for GnRH agonists to suppress menses. reduced intensity allogeneic HCT: A reduced-intensity conditioning transplant is a bone marrow or cord blood transplant (also called a BMT) that uses less intense treatment to prepare for transplant than a standard transplant does. While a standard transplant uses the pre-transplant treatment to destroy most of the disease cells, a reduced-intensity transplant relies on the donor's immune cells to fight disease.
Comparison of Luteinizing Hormone (LH) Levels	31.9 IU/L Standard Deviation 36.1	27.8 IU/L Standard Deviation 41.6

SECONDARY outcome

Timeframe: 2 years

Population: Four patients on the Observational arm never had any follow-up FSH levels drawn and were removed from this analysis. All 7 patients on the Interventional arm and 8 patients on the Observational arm were lost to follow-up.

Comparison of treatment arms; interventional versus observational average LH levels during study.

Outcome measures

Measure	Interventional - Received Leuprolide Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-HCT and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days for patients who undergo myeloablative allogeneic or autologous hematopoietic cell transplantation. Leuprolide: Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-transplant (HCT) and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days hematopoietic cell transplant: Conventional bone marrow transplant regimen.	Observational Arm n=2 Participants Women undergoing reduced intensity allogeneic HCT will be observed. Progestin contraceptives, Norethindrone acetate, and any other hormone methods can be used according to the prescription guidelines except for GnRH agonists to suppress menses. reduced intensity allogeneic HCT: A reduced-intensity conditioning transplant is a bone marrow or cord blood transplant (also called a BMT) that uses less intense treatment to prepare for transplant than a standard transplant does. While a standard transplant uses the pre-transplant treatment to destroy most of the disease cells, a reduced-intensity transplant relies on the donor's immune cells to fight disease.
Comparison of Leuprolide Hormone (LH) Levels	—	5.6 IU/L Standard Deviation 4.3

SECONDARY outcome

Timeframe: Day Prior to Transplant

Population: Six patients on the Observational arm never had any follow-up AMH levels drawn and were removed from this analysis.

Comparison of treatment arms; interventional versus observational average AMH levels after receiving transplant.

Outcome measures

Measure	Interventional - Received Leuprolide n=7 Participants Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-HCT and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days for patients who undergo myeloablative allogeneic or autologous hematopoietic cell transplantation. Leuprolide: Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-transplant (HCT) and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days hematopoietic cell transplant: Conventional bone marrow transplant regimen.	Observational Arm n=4 Participants Women undergoing reduced intensity allogeneic HCT will be observed. Progestin contraceptives, Norethindrone acetate, and any other hormone methods can be used according to the prescription guidelines except for GnRH agonists to suppress menses. reduced intensity allogeneic HCT: A reduced-intensity conditioning transplant is a bone marrow or cord blood transplant (also called a BMT) that uses less intense treatment to prepare for transplant than a standard transplant does. While a standard transplant uses the pre-transplant treatment to destroy most of the disease cells, a reduced-intensity transplant relies on the donor's immune cells to fight disease.
Comparison of Antimullerian Hormone (AMH) Levels After Transplant	0.6 ng/ml Standard Deviation 0.9	6.4 ng/ml Standard Deviation 7.7

SECONDARY outcome

Timeframe: Day 180 after Transplant

Population: Four patients on the Observational and 10 patients on the Observational arm never had any follow-up AMH levels drawn and were removed from this analysis.

Comparison of treatment arms; interventional versus observational average AMH levels after receiving transplant.

Outcome measures

Measure	Interventional - Received Leuprolide n=3 Participants Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-HCT and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days for patients who undergo myeloablative allogeneic or autologous hematopoietic cell transplantation. Leuprolide: Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-transplant (HCT) and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days hematopoietic cell transplant: Conventional bone marrow transplant regimen.	Observational Arm Women undergoing reduced intensity allogeneic HCT will be observed. Progestin contraceptives, Norethindrone acetate, and any other hormone methods can be used according to the prescription guidelines except for GnRH agonists to suppress menses. reduced intensity allogeneic HCT: A reduced-intensity conditioning transplant is a bone marrow or cord blood transplant (also called a BMT) that uses less intense treatment to prepare for transplant than a standard transplant does. While a standard transplant uses the pre-transplant treatment to destroy most of the disease cells, a reduced-intensity transplant relies on the donor's immune cells to fight disease.
Comparison of Antimullerian Hormone (AMH) Levels After Transplant	0.08 ng/ml Standard Deviation 0	—

Adverse Events

Interventional - Received Leuprolide

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Observational Arm

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Interventional - Received Leuprolide n=7 participants at risk Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-HCT and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days for patients who undergo myeloablative allogeneic or autologous hematopoietic cell transplantation. Leuprolide: Long-acting leuprolide 11.25 mg intramuscularly (IM) pre-transplant (HCT) and 3 months post-HCT PLUS Short-acting leuprolide 0.2 mg subcutaneously (SQ) daily for 14 days hematopoietic cell transplant: Conventional bone marrow transplant regimen.	Observational Arm n=10 participants at risk Women undergoing reduced intensity allogeneic HCT will be observed. Progestin contraceptives, Norethindrone acetate, and any other hormone methods can be used according to the prescription guidelines except for GnRH agonists to suppress menses. reduced intensity allogeneic HCT: A reduced-intensity conditioning transplant is a bone marrow or cord blood transplant (also called a BMT) that uses less intense treatment to prepare for transplant than a standard transplant does. While a standard transplant uses the pre-transplant treatment to destroy most of the disease cells, a reduced-intensity transplant relies on the donor's immune cells to fight disease.
Vascular disorders Hot Flashes	42.9% 3/7	0.00% 0/10
Psychiatric disorders Mood Liability	42.9% 3/7	0.00% 0/10

Additional Information

Dr. Angela Smith

Masonic Cancer Center, University of Minnesota

Phone: 612-626-8094

Email: smith719@umn.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place