Understanding Stem Cell Transplant

1. Understanding Stem Cell Transplant Autologous and Allogeneic

2. Its all the same thing! • Bone Marrow Transplant - BMT • Stem Cell Transplant – SCT • Peripheral Blood Stem Cell Transplant – PBSCT • Hematopoietic Cell Transplant – HCT • Cord blood transplant In general terms, we will refer to this process as stem cell transplant.

3. What diseases are treated with Stem Cell Transplant? • Acute and Chronic Leukemia • Hodgkin's Lymphoma • Non-Hodgkin’s Lymphoma • Myelodysplastic Syndrome • Myeloproliferative Neoplasms • Multiple Meyloma • Germ Cell Tumor • Myelofibrosis • Aplastic Anemia • Immune-deficiency disorders • Hemoglobinopathies, including Sickle Cell Anemia • And more…

4. What are Stem Cells? • Immature cells, made by the bone marrow, that grow into mature red blood cells, white blood cells, and platelets. • Red blood cells • Carry oxygen to all cells in your body • White blood cells • Fight infection • Platelets • Help your blood clot

6. What interrupts this system? • Certain diseases can disrupt this function of the bone marrow, causing the bone marrow to fail, produce excess of certain types of blood cells, or cause those stem cells to not mature. A stem cell transplant may help to treat these diseases.

7. What is a Stem Cell Transplant? • A Procedure that replaces diseased cells with healthy cells. • Stem cells are collected, either from a donor or the patient. • The patient then receives chemotherapy, with or without radiation therapy, to attack the cancer cells. • The collected stem cells are then infused into the patient’s bloodstream to restart their stem-cell production and function.

8. How does it work? • The 2 main components = Conditioning and Engraftment • Conditioning= first step • Treat the blood, bone marrow and/or immune system with chemotherapy, total body irradiation or both to destroy the malfunctioning cells. • This is done in the Hospital = Admission for Transplant • Engraftment = second step • Stem cells are infused into the patients bloodstream. • The stem cells migrate to the bone morrow where they grow and develop .

9. Conditioning – 2 types High-Dose Conditioning aka reduced-intensity, non-myeloablative – only used with AlloTransplant Designed to weaken, but not destroy the bone marrow and immune system so that the patients body will accept and not destroy donor cells. Often used for those who are older or have additional health problems Reduced-Dose Conditioning • aka myeloablative • Designed to eliminate malignant (cancerous) cells • Disable the immune system • Destroy the function of the bone marrow • This process eliminates the patients immune system and the ability to form new blood cells • “myelo” = bone marrow • “ablative” = to destroy • All Auto Transplants use High-Dose Conditioning.

10. EngraftmentDiffers based on type of conditioning High-dose conditioning Reduced-dose conditioning Engraftment occurs when the patient develops a new immune system alongside the patient existing immune system = a mixed immune system. Goal = new immune system will attack any remaining cancer cells (Graft-versus-tumor effect) and eventually take over. Only used with Allo Transplant • Engraftment occurs when the patients body resumes producing blood cells and develops an immune system • Remember all Auto’s use High-dose conditioning.

11. Types of Stem Cell Transplant • Your doctor will explain which type is recommended for you based on many factors, including your disease and the expected response. • The distinction between the types of transplant has to do with the source of the stem cells.

12. The Transplant Process It’s a long process and challenging journey. It will evolve and change along the way. We ask that you be flexible as this process can be unpredictable at times!

13. The Transplant Process • Plan ahead – organize, identify caregiver, housing if needed. • Preparation – insurance approval, medical eligibility, identify donor, stem cell mobilization and collection • Admission for transplant– Conditioning, Transplant, Engraftment, Discharge • Recovery • Long term survivorship

14. Planning Ahead - Caregiver • Identify a Caregiver: This is a person or group of people that will help to care for you throughout the transplant process. They play a very important role in the success of your transplant. In fact, it is such an important role that you MUST have a caregiver or group of caregivers in order to have a transplant at UCH. • Spouse, partner, adult child, parent, sibling or a friend who is willing to care for you. • If multiple caregivers will be taking care of you, a written schedule listing each persons time would be helpful to create prior to Transplant. • Your caregivers need to be available to you most of the time.

15. Planning Ahead - Caregiver • Why do I need a caregiver? For your safety. Caregiver responsibilities include the following: • Assistance with activities of daily living. • Transportation • Meal preparation • Cleaning • Medication management • Central line care • Emotional support • Seeking emergency medical assistance if needed. • And much more……More information on caregiver responsibilities can be found in the Bone and Marrow Transplant book.

16. Planning Ahead - Caregiver • Your caregiver does not need to stay with you while you are admitted to the hospital for transplant. Although they are welcome to be there 24/7 if you want them to be. • We do ask that your caregiver has some type of local lodging during this time to shower, do laundry etc. • Prior to discharge from the hospital we will provide your caregiver(s) with information/instructions for what precautions to take when you return to home or to local lodging. • Discharge information can also be found in the UCH Blood & Marrow Transplantation book.

17. Planning Ahead - Caregiver • The caregiver role can be very demanding. It is important that your caregivers also stay healthy and seek support. Our social workers and psychologist have resources available to caregivers if needed. • More information on caregiver support can be found in the Bone and Marrow Transplant book.

18. Planning Ahead: Lodging • Upon discharge from the hospital for transplant, it is required that you live/stay within a 30 minute drive from the hospital for your safety. • If you have problems or questions regarding local housing arrangements, please contact one of our social workers. • Some insurance providers offer travel and lodging benefits for patients who travel beyond a certain distance. Our Transplant Financial Case Coordinators can find out if this is an option for you. • It is your responsibility to determine how to access them by contacting your insurance company.

19. Planning Ahead: Lodging • There is a list of lodging options that are close to UCH in the back of the Blood and Marrow Transplant Book. • We advise that you or someone on your behalf checkout the lodging options in advance. • The University of Colorado Hospital dose not inspect or approve these facilities and therefore cannot assume any responsibility for the accommodations or charges.

20. Planning Ahead – How long do I need to stay locally with a caregiver?

21. Planning Ahead: Miscellaneous Items • Advanced Directives – Documents in which you either state your choices for medical treatment or designate who should make treatment choices if you cannot make decisions. The 2 types of advanced directives are Living Will and Medical Durable Power of Attorney.

23. Planning Ahead: Miscellaneous Items • FMLA Paperwork - is a United States federal law requiring covered employers to provide employees job-protection and unpaid leave for qualified medical and family reasons. If you need assistance with this paperwork, please contact your coordinator or a social worker. • Fertility Options – Chemotherapy and Transplant can cause infertility. If you have any questions regarding fertility options, please discuss this with your Oncologist. We can make a referral to see a OncoFertility Specialist.

24. Planning Ahead: Miscellaneous Items • Smoking Cessation - For transplant we require that you do not smoke any form of Tobacco or Marijuana. If you would like more information on Smoking Cessation, please contact one of our psychologists. • Dental Care – The mouth can be a source of infection following transplant. You should receive a thorough dental examination and cleaning prior to transplant. - Some insurance companies require this prior to transplant. - See Handout

26. Prepare your friends and family! People want to know how they can help. Maintenance of your home, yard, assistance with paying bills on time, caring for pets and children, etc.

27. Preparation: Insurance Approval • Insurance approval is required for both the pre-transplant workup and the transplant itself. • The Transplant Financial Case Coordinators (TFCC’s) will submit these requests to the insurance company. • It is required that all transplant patients have a payer source. • Insurance companies vary in terms of requirements. • Medicare A &B – required to have a secondary payer source. • Every patient will be assigned a Case Manager at the insurance company. • NOTHING CAN BE DONE IN REGARDS TO TRANSPLANT WITHOUT INSURANCE APPROVAL!

28. Preparation: Medical Eligibility • After insurance approval has been received, you will be required to undergo a pre-transplant workup to further determine your eligibility for transplant. Eligibility for transplant requires that you are physically able to undergo a transplant. • The timing of this pre-transplant workup will vary, but in general, it will be done within 30 days of your scheduled transplant. • Your coordinator will provide you with a very specific schedule for the pre-transplant workup.

29. Preparation: Medical Eligibility • For an explanation of what tests will be done at the pre-transplant workup, please see the Blood and Marrow Transplant book. (PET, CT, Skeletal Survey, MRI, CXR, ECHO, PFT’s, Infectious Disease testing, labs, Psych, Consent for apheresis, • *** If there is a delay for any reason, that causes these tests to fall outside of the 30 days before transplant, these tests may need to be repeated ***

30. Preparation: Selecting a Donor • Your physician will discuss this with you which donor source will be used for your transplant. • Cord Blood Transplant – done using stem cells from an umbilical cord donated by a mother right after the babies birth. • Matched Unrelated Donor (MUD) –a donor from the general population who is not related to you but who is a very close match genetically. • If you have friends or family members that wish to register to become a potential donor, they can go to the National Marrow Donor Program and Be The Match Foundation’s website at www.marrow.org.

31. Preparation: Stem Cell Collection • After the donor source has been identified, the next step is to Mobilize and Collect the stem cells from the donor source. • Where do the stem cells come from? • Bone Marrow: This is the soft, spongy tissue found inside of our bones Stem cells live in this tissue. They can be collected from the bone marrow through a procedure called Bone Marrow Harvest. • Peripheral Blood: Stem Cells sometimes leave the bone marrow and circulate in the peripheral bloodstream. Normally there are very few stem cells in the blood stream. They can be collected from the peripheral blood stream through a procedure called Apheresis. • Umbilical cord Blood: At birth, there is a large amount of stem cells circulating within the placenta and umbilical cord. They are collected at that time, frozen and stored.

32. Preparation: Stem Cell Collection • How do we get stem cells out of the bone marrow or peripheral blood stream? • Bone Marrow Harvest: This type of stem cell collection is done in the operating room and requires general anesthesia. Special needles are used to remove bone marrow from the pelvic bone. This method is rarely used. • Apheresis:This is the most common type of stem cell collection. It is done by drawing blood out of the donor through a catheter into a machine that separates the stem cells out of the blood stream. The stem cells are collected and the rest of the blood is returned to the donor. • Autologous patients: Takes 1-5 days, 4-6 hours per day. • Allogeneic donors: Takes 1-2 day, 4-6 hours per day.

33. Preparation: Stem Cell Collection • Normally there are very few stem cells circulating in our peripheral bloodstream at any point in time. Remember that stem cells live INSIDE the bone marrow. • How do stem cells get out of the bone marrow and into the blood stream where they can be collected using apheresis? • Through the use of medications, stem cells can be forced to leave the bone marrow and circulate in the blood stream. This process is called Mobilization. • Typically it takes a few days after receiving the medications for the stem cells to mobilize • The Medications used to do this are Neupogen and Mozobil.

34. Preparation: Stem Cell Mobilization • Neupogen – an injection given daily for 4-10 days for autologous patients and 4 days for donors. It stimulates the growth of white blood cells. • It is given at high doses to overstimulate the bone marrow to make white blood cells. • The over production of white blood cells causes overcrowding in the bone marrow space. • This results in stem cells leaving the bone marrow and entering the blood stream where they can then be collected through apheresis. • Mozobil – an injection given the night before the apheresis procedure. • Stimulates the stem cells to actively leave the bone marrow and enter the peripheral blood stream. • Not always used

35. Preparation: Stem Cell Mobilization • These injections can be given at home by you or a caregiver. If given at home, you or a caregiver will be instructed on how to administer the injection correctly. • In some instances your insurance may require that you come to the Bone Marrow Transplant Infusion Center (BIC) for the injections daily. • Your nurse coordinator will contact the insurance company to determine if the Neupogen and Mozobil need to be administered at the BIC or at home.

36. Preparation: Stem Cell Collection • Central Line. • A Central Line, also known as a Trifusion Line will be placed prior to the start of collection. This line will be used for the apheresis procedure and stem cell transplant. It will remain in place for the entire transplant process. • Most donors do not need a central line for collection. In most cases, 2 large peripheral intravenous catheters will be placed in the arm. If peripheral access can not be obtained, a temporary central line will be placed. • You and your caregiver will be provided education on how to care for your central line at home by a RN in the Bone Marrow Transplant Infusion Center.

37. Trifusion Line – A central line that is used for stem cell collection. It will also be used during the stem cell transplant.

38. Preparation: Stem Cell Collection • Matched Unrelated Donorsare usually from other states or countries. For donations occurring outside the University of Colorado Hospital, the cells are delivered via courier. • Cord Blood units are already collected. Therefore they are shipped to UCH just before the transplant admission.

39. Preparation: Stem Cell Collection • How to prepare for the Apheresis procedure. • Arrive at 7:30am. A lab check may or may not be done prior to starting collection. • Eat breakfast before you arrive for the procedure. • Bring snacks/lunch, you will not be able to leave at any point while attached to the apheresis machine. • It is best to have a caregiver with you for this reason. • Bring something to do – reading material, movies, headphones etc.

40. Preparation: Stem Cell Collection • Your nurse coordinator will give you a specific schedule and instructions on how to prepare for stem cell collection for both autologous patients and related donors. • The timing of stem cell collection will vary depending on the type of disease, type of transplant, and the type and timing of other chemotherapy treatments…. • Please Remain Flexible –stem cell mobilization and collection can be very unpredictable. In some cases, it has to be repeated. If there are any changes in the plans, a member of the BMT healthcare team will discuss this with you at that time.

41. Example Collection Schedule

42. Example Collection Schedule

43. The Apheresis machine and one of our Apheresis RN’s

44. Where are we so far? • Planned ahead – Caregiver, Lodging etc.. • Insurance Approval • Medical eligibility confirmed through pre-transplant workup • Donor is selected (Autologous, Sibling, Matched Unrelated or Cord Blood) • Central line Placement • Stem cell mobilization and collection complete • What is next???

45. Transplant – How long will you be in the hospital? Remember the time frame to engraftment is different for every patient.

46. Transplant Admission • For information on what to bring to the hospital please see the Blood and Marrow Transplant book. • Upon admission, a central line will be placed if you do not already have one. • The Conditioning Chemotherapy/Radiation Treatment will be started. • The type of conditioning chemotherapy you received is based on the type of disease, type of transplant, medical condition and many other factors. This will be determined prior to admission and will be discussed between you and your attending physician.

47. Example Conditioning Regimen – RBEAM – Autologous Transplant

48. Example Conditioning Regimen – Melphalan - Autologous Transplant

49. Example Conditioning Regimen – FLU- 2Gy TBI – Allogeneic Transplant

50. Example Conditioning Regimen – Bu-Cy-Allogeneic Transplant