Fertility Preservation in Women Who Will Have Gonadotoxic Therapy or Hematopoetic Stem Cell Transplantation, and in Women With Sickle Cell Disease
Condition: Hemoglobin; Stem Cell Transplant; Aplastic Anemia
MeSH terms: Anemia, Sickle Cell; Anemia, Aplastic
Study type: Observational

Summary: 
      Background:

      - Some treatments for cancer or other diseases can lead to infertility in women. These
      treatments include chemotherapy, some stem cell transplants, and pelvic radiotherapy. They
      are called gonadotoxic therapies. Women can now have their eggs frozen before they have these
      treatments. This may allow them to get pregnant later. Researchers want to learn more about
      this technology and processes.

      Objectives:

      - To provide egg freezing for women having gonadotoxic therapies at NIH. To learn more about
      the effects of these therapies.

      Eligibility:

      - Women at least 18 years old who are past puberty and before menopause. They must be
      scheduled to have gonadotoxic therapies.

      Design:

        -  Participants will be screened with medical history and blood and hormone tests. They
           will also have a physical exam and transvaginal ultrasound.

        -  Ovary stimulation: participants will have medications injected under the skin. These
           increase the chance of fertility. This phase will take about 8 20 days. Participants
           will have blood drawn and transvaginal ultrasound daily or every other day. Some
           participants will also have blood thinner injected daily.

        -  Egg retrieval: participants will check in to the hospital. Eggs will be removed with a
           needle during a short surgery. Participants will be awake but sedated.

        -  Participants may stay overnight in the hospital.

        -  They will return every 1 3 days for 1 3 weeks for blood tests.

        -  Mature eggs will be frozen after egg retrieval and immature eggs (which cannot be
           fertilized for clinical use) will be used for research. Participants can use their eggs
           in the future at outside, private fertility clinics to try to become pregnant. If the
           eggs are stored for more than 5 years, participants must pay for storage.
    

Detailed description: 
      Treatment with chemotherapeutic drugs, hematopoietic stem cell transplantation, and pelvic
      radiotherapy for cancer or other serious medical illnesses has the potential to markedly
      increase the risk of gonadotoxicity leading to infertility in women. Females who are
      post-menarchal with these risk factors may be candidates for fertility preservation through
      oocyte cryopreservation before ovarian failure ensues. For example, sickle cell disease (SCD)
      is the most common hemoglobinopathy in the United States (3). Hypoxic conditions cause the
      abnormal hemoglobin molecule to undergo sickling which leads to painful microvascular
      occlusion. SCD is associated with multiple organ system dysfunction as well as neurological
      and pulmonary complications, which can lead to early mortality. Hematopoietic stem cell
      transplantation (HSCT) is the only treatment currently available for SCD that results in a
      complete cure. In patients who have undergone HSCT with a matched sibling, event-free
      survival has been as high as 85%-95%. Multiple studies have unfortunately demonstrated that
      infertility and premature ovarian insufficiency are quite common following HSCT. Specifically
      in our patient population with sickle cell disease, we have recently found largely preserved
      ovarian function prior to transplantation, but profound gonadotoxicity following transplant
      (unpublished). This underscores the clinical need for additional, effective fertility
      preservation methods for our at-risk populations.
    

Observational model: Cohort
Time perspective: Prospective
Keywords: Bone Marrow Transplant; Fertility; Ovary