Texas native John Vasquez led an active life until he started to experience unusual symptoms in 2015, including an incidence of numbness on one side of his body. The 20-year-old was soon diagnosed with aplastic anemia aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… .
He sought local treatment and was started on immunosuppressive therapy immunosuppressive therapy: Immunosuppressive drug therapy lowers your body's immune response. This prevents your immune system from attacking your bone marrow, allowing bone marrow stem cells to grow, which raises blood counts. For older patients with acquired aplastic anemia, immunosuppressive drug therapy is the… , causing him to suffer the full range of side effects – fever, chills, nausea and serum sickness serum sickness: An immune system reaction to foreign proteins in certain medicines. Serum sickness can be a side effect of ATG, causing fever, rash, joint pain, and muscle aches. , which includes joint and muscle pain. Rather than wait for his doctors’ next steps, John decided to contact the National Institutes of Health (NIH) himself. He was eventually approved for a stem cell transplant as part of a new clinical trial clinical trial: A type of research study that tests how a drug, medical device, or treatment approach works in people. There are several types of clinical trials. Treatment trials test new treatment options. Diagnostic trials test new ways to diagnose a disease. Screening trials test the best way to detect a… testing the use of cord-blood stem cells stem cells: Cells in the body that develop into other cells. There are two main sources of stem cells. Embryonic stem cells come from human embryos and are used in medical research. Adult stem cells in the body repair and maintain the organ or tissue in which they are found. Blood-forming (hemapoietic) stem… with half-matched stem cells from his brother, Isaiah.
Although this approach has previously been tested with other diseases, John became the very first aplastic anemia patient admitted for this protocol protocol: An action plan that describes what will be done in a clinical trial and how it will be carried out. This plan is reviewed and approved by a committee at each place doing the clinical trial. This committee is known as the Institutional Review Board. , making him the so-called "first in human" test of this particular kind. Study leader Dr. Richard Childs was very pleased with John’s transplant results. "We’re very excited to find that our study combining mismatched, expanded cord blood units and half-matched donor stem cells from a family member had survival rates comparable to those from the fully-matched related donors."
Preparation and Recovery
John and his sister/caregiver Isabella journeyed to NIH from their hometown of San Antonio in May 2017 so he could begin the chemotherapy chemotherapy: (kee-moe-THER-uh-pee) The use of medicines that kill cells (cytotoxic agents). People with high-risk or intermediate-2 risk myelodysplastic syndrome (MDS) may be given chemotherapy to kill bone marrow cells that have an abnormal size, shape, or look. Chemotherapy hurts healthy cells along with… and radiation treatments that are required to prepare a patient for transplantation. With most of his family by his side, the stem cell transplant occurred on August 11. If everything goes according to plan, John and his sister could be back home by February of next year.
We spoke with John about a month after his transplant. "At first there was a lot of pain from the transplant conditioning, and it really took around a week and a half for this pain to recede. But I am getting back to some physical activity – even small things like getting out of the chair or bed and walking around the hospital floor is considered progress. Each day, I feel a little bit better and more alert."
John’s medical team taught Isabella how important it is to rebuild her brother’s strength through activity. "The whole idea is to get him physically and mentally active again in this immediate post-transplant period. I have to push him to the point that’s allowed for his condition, but it’s essential for his recovery," she told us. "I have to make sure he’s meeting the goals that are set, and let our team know if he isn’t. But they say that he’s coming along well!"
Slow but mostly steady
John has been residing on the NIH campus since May but has mostly been staying at the NIH Children’s Inn following the transplant. That’s where families participating in the Institute’s cutting-edge treatments are provided with comfortable housing. Moving from inpatient status to an outpatient facility marked a new phase in John’s post-treatment life.
It’s not uncommon for patients in this phase to return to the hospital setting from time to time for extra care. Their immune systems are still delicate, and every new development during a patient’s recovery gets a rapid response to these changes. John, for example, was moved back to the hospital for two weeks of treatment following a minor infection.
His sister says that, "As a caregiver, I’m relieved when John is back in the outpatient setting, so I don’t have to worry about what might be happening to him when he’s there and I am here."
John has a different point of view. "Inpatient days have a set routine. Everything comes to you – the medication schedule occurs at a certain time and most other events and appointments are very structured. As an outpatient, my days seem to run together and are more tiring because of the physical activity required to just get around and do what needs to be done. I still have to go back to the hospital for certain things two or maybe three times a week."
John reports that it can be hard to find the energy he needs after being so sedentary as an inpatient, but his sister remains a guiding light. In addition to loving support, she helps her brother stay active and keeps track of his complex medication schedule. "I try to increase his activity duration by small increments, around 5 minutes each day, and I make sure he’s eating properly, getting enough fluids and taking the medicines."
She also cherishes the chance to get him out of the facility now and then. Even though he always has to wear a mask, Isabella likes to take her brother on short shopping trips with other patients and caregivers staying at the Inn. While these little excursions may result in getting a few staples, they are more about patients and families who long to begin the process of normalizing their lives, one day at a time.