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Caring for Your Child During Gene Therapy Program for Cell and Gene Therapy
Caring for Your Child During Gene Therapy
Hematology/OncologyProgram for Cell and Gene Therapy
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Your Child’s Information
Patient’s name
Date of birth
Seattle Children’s Hospital medical record number
Date of diagnosis
If found, return to (name and phone number):
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Caring for Your Child During Gene Therapy
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Contents
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ContentsYour Cellular and Gene Therapy Team ............................................................7
Consent (insert)
What to Expect
What to Expect During Cellular and Gene Therapy ................................9
Your Child’s Healthcare Team .............................................................................15
Free Interpreter Services ......................................................................................17
Parents and Caregivers: Emergency Steps/Chickenpox ..................... 19
Temperature Conversion Chart ........................................................................20
Blood Counts
Infection Signs and Blood Counts - PE916 ..........................................Insert
Recording Your Child’s Blood Counts ............................................................21
Resources
Resources .................................................................................................................... 27
Glossary ........................................................................................................................29
InsertsGeneral Anesthesia and Your Child - PE620Transfusion Instructions: Signs to Watch For After a Blood Transfusion - PE661Acute Pain - PE503Assessing Children’s Pain: Pain Intensity Assessment Tools - PE952Chlorhexidine Wipes - PE1327Keep Germs Away From Your Child - PE454Help Prevent Catheter- Associated Bloodstream Infections - PE1059Types of Central Lines and Their Care - PE2062Caring for a Hickman or Broviac Central Line Catheter - PE1091NG Tube Feeding Instructions - PE1699Places to Stay - PI600Transportation - PI602Restaurants - PI601Family Resource Center - PI198Activities for Families - PI599Guest Services - PI749Directions To and From Ronald McDonald House - PI719
Notes ..............................................................................................................................33
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Caring for Your Child During Gene Therapy
Your Cell and Gene Therapy Team
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Your Cell and Gene Therapy TeamHere are the healthcare providers of Seattle Children’s Program for Cell and Gene Therapy. All children having cellular and gene therapy may receive care from anyone on this team.
Immunologists and Bone Marrow Transplant SpecialistsImmunologists are doctors and scientists who investigate the immune system and treat patients with immune system disorders.
Bone Marrow Transplant Specialists are doctors who specialize in bone marrow transplants for patients with a variety of disorders.
Our doctors have specialty training in adult and pediatric immunologic disorders, including primary immunodeficiency disorders. They are known around the world for their research on new ways to diagnose, treat and cure Primary Immunodeficiencies. Seattle Children’s is one of the few places in the world where doctors are both conducting research and caring for patients.
Dr. David
Rawlings
Dr. Hans Ochs Dr. Andrew
Scharenberg
Dr. Suzanne
Skoda-Smith
Dr. Troy
Torgerson
Dr. Lauri
Burroughs
Dr. Anne
Woolfrey
ARNP Kathey
MohanDr. Aleksandra
Petrovic
Dr. Kanwal
Mallhi
Dr. K. Scott
Baker
Caring for Your Child During Gene Therapy
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Your Research Team In addition to your physicians, you will have research team members monitoring your child’s progress on the research protocol. A clinical research nurse is a nurse with expertise in research and treatment protocols for your child and their immune deficiency. Our research nurse is a point of contact for our patients on protocols and provides education, answers many ongoing questions, and coordinates follow-up related to the research protocol on which your child is receiving treatment. The research nurse is a good person to call when you’re not sure who to call for continued research needs.
A clinical research associate, or CRA, is a specialized research professional who will ensure your care is followed according to the protocol that you have consented to. Our CRA will assist in coordinating follow-up and making sure specific time points are met in assessing your progress on the treatment protocol. The CRA ensures protocol and regulatory compliance throughout the clinical trial.
Social WorkersSocial workers are skilled counselors who give families emotional support, from diagnosis through care and recovery. They coordinate care between families, the community and the medical team; and help families get the resources they need. Please let your provider know if you would like to meet with a hospital social worker.
Kathleen Teaderman, RN Tara Bumgarner, CRA
What to Expect During Cellular and Gene Therapy
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What to Expect During Cellular and Gene Therapy What is cellular and gene therapy?Most primary immune disorders are caused by errors in specific genes. During cellular and gene therapy, a person’s own stem cells are treated to correct the genetic defect in the cell, and then the cells are reintroduced into the patient through an IV. (Please refer to the glossary at the end of this guide to aid in understanding of key terms.)
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Where does my child go for cellular and gene therapy care?Care of your child, adolescent or young adult undergoing gene therapy takes place in four locations:
Immunology ClinicThis is the first place you will go after the initial diagnosis of your child. You and your child will come to this clinic for planned visits with the doctor and potential lab tests before starting the gene therapy process. The clinic phone number is 206-987-7450. Prior to transplant, please contact your pediatrician for concerns about the health of your child. Your pediatrician will be in close contact with our immunology specialists.
Cancer Care UnitChildren are admitted to the Cancer Care Unit for several situations during the process of treatment. Your child may be admitted to the Cancer Care Unit for the following reasons: • Your child becomes ill before their gene therapy and your doctor feels it’s
a necessary safeguard to prevent further infection. • Your child is better served by the protective isolation provided by being
in the hospital.• Your child will be admitted to this unit for the entirety of the gene
therapy process. • Your child becomes ill once home after their gene therapy hospitalization
and the SCCA clinic is closed.
The unit is open 24 hours a day. The reception desk phone number is 206-987-2032. Both parents may sleep at the bedside and visitors are allowed from 8 a.m. until 8 p.m. Infection control guidelines are strictly monitored for the safety of all of our patients. A nurse will cover unit- specific guidelines at the beginning of each admission. Please feel free to ask any questions that you might have.
Pediatric Intensive Care Unit (PICU)This unit is for children who require closer attention due to worsening symptoms. For example, a child may get transferred to this unit if they need special medicine to keep their blood pressure up or a breathing tube to help them breathe.
What to Expect While Receiving Cellular and Gene Therapy
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SCCA Outpatient Clinic Treatment-related outpatient care for children undergoing bone marrow transplantation and gene therapy occurs at the SCCA outpatient clinic on Lake Union. Once cellular and gene therapy is determined to be the best route for your child’s treatment, you will begin to take your child to the SCCA clinic for care, including care you were previously receiving from the doctors at the Immunology Clinic. The SCCA Clinic is at 1354 Aloha St., Seattle, WA 98109. Your child will be seen by members of the “Green” or pediatric team who specialize in pediatric care. The phone number to call here is 206-606-7600 for RN assistance and triage needs. Please call if your child has a fever or if you have other concerns about your child’s health.
Signing the consentOnce your child has a confirmed diagnosis of a primary immune disorder and is found eligible for gene therapy, you and your child will have your initial or “intake” visit. This visit includes members of the Bone Marrow Transplant team and the Immunology team. At this visit you will be given consent to sign and an opportunity to ask questions of all of your child’s providers.
Starting the treatmentAt the start of their treatment, your child will have several procedures that require sedation, or being put to sleep. • Central line — One of the treatments is placement of a Central Venous
Catheter (CVC), or central line. This is a small, flexible tube inserted into a large vein near the heart. This serves as a route to give medicine and fluids, and to get blood samples.
• Bone marrow aspirate — During a bone marrow aspirate, your child is taken to an operating room or special procedure room where providers obtain a sample of bone marrow using a needle and syringe. This is done to obtain a sample for laboratory study. Please refer to the glossary at the end of this book for detailed information about procedures, terms and topics like these.
On a separate day, your child will undergo hematopoietic stem cell collection for the gene therapy procedure. This will happen in one of two ways:• Bone marrow harvest — In the operating room, cells are collected from
your child’s hip bones with a needle and syringe.• Peripheral blood stem cell apheresis — Whole blood is removed from
your child through an IV, and a centrifuge separates the peripheral blood stem cells from other components. The other components are returned to your child through an IV.
After the doctors collect the stem cells from your child, the cells are taken to a special laboratory where they are treated to have the desired gene. This process takes about four days, and the timing is closely monitored by all staff involved in the care of your child.
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Caring for Your Child During Gene Therapy
Your child will be admitted to the Cancer Care Unit after the hematopoietic stem cell collection for monitoring and will start chemotherapy, or “conditioning.” • Chemotherapy — Chemotherapy is a medicine that is given through your
child’s central line. The chemotherapy gets rid of some of the existing cells in the bone marrow to make room for the gene-treated cells growing in the laboratory. During chemotherapy, several blood draws may be done to monitor the levels of the medicine in your child. The labs (blood draws) will usually be drawn through your child’s central line.
The infusion — treated cells returned to your child’s bodyWhen the cells have been treated and closely monitored at the laboratory for the correct number and genetic makeup, the cells will be returned into your child’s vein during the hospital stay. After chemotherapy is finished these new cells are given to your child through the central line in their hospital room on the Cancer Care Unit. Putting cells in through an IV is called an infusion. Your child will be closely monitored during the infusion by both doctors and nurses. Medicines to prevent reactions will also be given. Although the infusion of stem cells is a brief procedure, it is a big event!
Engraftment or immune reconstitution — growing new cells inside the bodyAbout 10 to 28 days after infusion, new stem cells start to grow (engraft). Blood counts are watched daily as the graft strengthens. Often the first sign of a graft is a rising white blood cell count.
You can keep a daily record of your child’s blood counts to track progress. Forms are in this handbook. Your nurses can provide daily printouts of your child’s cell counts on request. In addition to watching for the first signs that the new immune system is working, staff are monitoring your child very closely to detect any early signs of complications so they can begin treatment promptly if needed.
The hospital stayYour child will remain in the hospital until they are medically stable and ready to be discharged. This time will vary. It depends on how your child’s immune system progresses, whether or not they need intravenous medications, and other medical considerations. During your child’s hospital stay, they will receive standard medical care for any medical problems that arise, either as a result of their immunodeficiency or as a result of the gene transfer. For instance, if your child develops infections due to lack of immune function, the infections will be treated with medicines that are commonly used to treat these infections. If your child has problems with keeping food down or getting nourishment, nutritional supplements
What to Expect While Receiving Cellular and Gene Therapy
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will be given. Many of these treatments are standard for children with an immunodeficiency. If complications arise due to the gene transfer, these will be treated with the best available treatments.
Recovery after infusionThe recovery of the immune system may take as long as 3 to 4 months after the cell infusion. If your child does not recover immune function within 15 to 17 weeks after the gene transfer procedure, they will be offered another form of stem cell transplant.
Hospital staff will be monitoring your child for any signs of complications, but you as the caregiver know your child best. Please feel comfortable sharing any concerns you may have with anyone on your care team.
Possible complications• Viral, bacterial, and fungal infections. These can be serious problems after
a transplant. Most infections can be successfully treated with medicine.• Pneumonia. This may occur in the first months after transplant. Your
child will have regular chest X-rays, and we will watch for early signs of pneumonia.
• Sinusoidal Obstruction Syndrome (SOS). This is a complication in which the liver’s job of removing waste products from the body is impaired due to the chemotherapy medicines. If this occurs, it will most likely occur during the first month after transplant. We will give your child medicine to prevent SOS.
Recovery after engraftmentYour child will be discharged from the Cancer Care Unit once they have demonstrated immune reconstitution or engraftment and after other discharge criteria are met. If your child has a continued need for their central line, we will provide training for you to care for this before you go home. We will help arrange for home care company options for supplies.
We will need to monitor your child frequently for any complications and recovering immune function. You and your child will come for outpatient visits to the SCCA outpatient clinic on Lake Union or the Immunology Clinic at Seattle Children’s. Your care team will schedule your initial follow-up appointments that will include visits with your doctors and research nurse, potential lab draws and nutrition checks.
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Caring for Your Child During Gene Therapy
Your Child’s Healthcare Team
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Your Child’s Healthcare TeamNames and phone numbers you will need — write them in the spaces below.
Attending MD
Fellow MD
Nurse practitioner Voicemail
Social worker Voicemail
Child Life specialist Voicemail
Dietitian Voicemail
Appointment scheduler/Patient care coordinator (PCC)
Call 206-987-7450 and press 1.
Other useful numbers
Hospital paging operator (main switchboard)
If you need to call after hours, use this number to reach the Immunology provider on call.
206-987-2000
Immunology Clinic 206-987-7450
Inpatient Cancer Care Unit 206-987-2032
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Ronald McDonald House 206-838-0600
Children’s Outpatient Pharmacy 206-987-2138
Children’s Home Care 425-482-4000
Children’s Hospital toll-free number 1-866-987-2000
Other important numbers
Name Pager Voicemail Fax number
Ask healthcare providers who care for your child for their business cards. Keep them in a handy place.
Caring for Your Child During Gene Therapy
Free Interpreter Services
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Free Interpreter ServicesCalling the hospitalIt is very important that you are able to call the hospital and talk to your child’s nurse practitioner or doctor if you have questions or if your child is sick.
The hospital has an interpreter service that will call the hospital for you. You can call this number 24 hours a day, 7 days a week, for both urgent and non-urgent calls. Call the interpreter service at 1-866-583-1527.• Press 1 for Spanish• Press 2 for Somali• Press 3 for Vietnamese• Press 4 for Russian• Press 5 for other languages
For emergencies, call 911.
At the hospitalIf your child is in the hospital or visits the clinic, we can provide an interpreter to help you talk with your child’s doctors and nurses. We also use telephone interpreters if it is hard to get an interpreter to visit with you.
Servicios gratis de interpretaciónLlamando al hospitalEs muy importante que usted pueda comunicarse al hospital para hablar con su médico o la enfermera especializada si tiene preguntas o su niño está enfermo.
El hospital cuenta con un servicio de interpretación telefónica que le puede asistir con las llamadas al hospital sin costo alguno para usted.
Puede usar este número las 24 horas del día, 7 días a la semana para llamadas urgentes y no urgentes. El número para el servicio de interpretación telefónica es 1-866-583-1527.Para emergencias llame al: 911.
Estando en el hospitalSi su niño está hospitalizado o tiene citas en las clínicas, nosotros podemos proveer servicio de intérpretes para ayudarle a comunicarse con los doctores y enfermeras de su niño. En caso de no conseguir un intérprete en persona dentro de un lapso de tiempo razonable, también contamos con servicios de interpretación telefónica.
Caring for Your Child During Gene Therapy
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Бесплатные переводческие услуги Звонки в госпитальОчень важно, чтобы Вы могли позвонить в госпиталь и поговорить с Вашей практикующей медсестрой или врачом, если у Вас есть вопросы или Ваш ребенок заболел.
В госпитале есть телефонная линия, обслуживаемая переводчиками, которые помогут Вам позвонить в госпиталь без каких-либо затрат с Вашей стороны.
Вы можете звонить по этому номеру круглосуточно, семь дней в неделю как по срочным, так и несрочным вопросам. Звоните в переводческую службу по тел. 1-866-583-1527.
Для получения неотложной помощи звоните по тел. 911.
Звонки внутри госпиталяЕсли Ваш ребенок находится в госпитале или на приеме в клинике, мы можем предоставить переводчика, чтобы помочь Вам общаться с врачом или медсестрой Вашего ребенка. Мы также пользуемся переводом по телефону, если переводчик не может придти лично.
Dịch Vụ Thông Dịch Miễn Phí Anh Gọi tới bệnh việnĐiều rất quan trọng là làm sao quý vị có thể gọi tới bệnh viện và nói chuyện với y tá thực hành hoặc bác sĩ của mình nếu có thắc mắc hoặc nếu con quý vị bị bệnh.
Bệnh viện có dịch vụ thông dịch qua điện thoại miễn phí để hỗ trợ quý vị khi quý vị cần gọi bệnh viện.
Quý vị có thể gọi cho dịch vụ thông dịch theo số 1-866-583-1527, 24 giờ một ngày, 7 ngày một tuần, cả cuộc gọi khẩn cấp hay không khẩn cấp.
Đối với những trường hợp cấp cứu, hãy gọi 911.
Ở bệnh viện Nếu con quý vị đang nằm bệnh viện hoặc đến khám tại phòng mạch, chúng tôi có thể sắp xếp thông dịch viên đến giúp quý vị nói chuyện với bác sĩ và y tá của con mình. Chúng tôi cũng sử dụng phiên dịch qua điện thoại nếu không thể sắp xếp cho thông dịch viên không có mặt ở đó.
Parents and Caregivers: Emergency Care Steps
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Parents and Caregivers: Emergency Care StepsCall 911 if you are worried that something is seriously wrong or if your child experiences any of the following:• Seizures, or is unconscious, unarousable or very confused• Not breathing, choking, or having severe difficulty in breathing• Severe, uncontrolled bleeding
Allergic reactionsThe signs of an allergic reaction include:• Hives – raised bumps on skin that usually itch• Itching• Redness of skin• Rash• Swollen tongue or lips – call 911• Difficulty breathing or swallowing – call 911
Signs of an allergic reaction can be an emergency. If your child has any of these signs, call the clinic or on-call doctor right away. If your child has swollen lips or tongue, or any difficulty breathing, call 911.
For all other concerns• Between 8 a.m. and 10 p.m. on weekdays, 8 a.m. and 6 p.m. on weekends,
and 8 a.m. and 5 p.m. on holidays, call the SCCA at 206-606-7600.• For all other times, call 206-987-2000 and ask for the Immunology
provider on call.You will be instructed about who to call at different points throughout your treatment. It is a good idea to keep your discharge instructions from each inpatient visit in this handbook.
Exposure to chickenpox or shinglesIt is very important for children with immune suppression to avoid being exposed to chickenpox or shingles. If you know that your child has been around someone with chickenpox, you need to call your child’s doctor right away. You also need to call your child’s doctor right away if you see a rash on your child that you think might be chickenpox or shingles.
Please call your healthcare provider to tell them of your concerns before bringing your child into any of the healthcare locations.
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Caring for Your Child During Gene Therapy
No Tylenol, ibuprofen (Advil or Motrin), or aspirin unless doctor approves each time.
Temperature Conversion Chart
°F °C Directions for patients
96.897.898.098.298.498.698.899.099.2
36.036.536.736.836.937.037.137.237.3
Normal temperature
99.499.699.8100.0100.2
37.437.637.737.837.9
Recheck temperature in 30 to 60 minutes
100.4100.6100.8
38.038.138.2
Call the clinic or on-call doctor for a temperature this high for an hour or longer.
101101.2101.4101.6101.8102.0102.2102.4102.6102.8103.0103.2103.4103.6103.8104.0104.2104.4104.6
38.338.438.638.738.838.939.039.139.239.339.439.639.739.839.940.040.140.240.3
Call the clinic or on-call doctor for fever this high or any higher.
Blood Counts
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Recording your child’s blood counts
Lab results recording chart
HCT PLT WBC ANC
Abbreviations:
HCT: Hematocrit
PLT: Platelets
WBC: White blood cells
ANC: Absolute neutrophil count
PRBC: Packed red blood cell transfusion
Normal values
31% to 48%
150,000 to 500,000
5,000 to 12,000
Over 1,000
Function Oxygen-carrying cells
Blood-clotting cells
Infection- fighting cells
Infection- fighting cells
Give PRBCs when Hct is less than 20%
Give platelets when child’s PLT is less than 10,000 to 30,000
Date HCT PLT WBC ANC Chemotherapy Other (transfusions, etc.)
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Caring for Your Child During Gene Therapy
Recording your child’s blood counts
Lab results recording chart
HCT PLT WBC ANC
Abbreviations:
HCT: Hematocrit
PLT: Platelets
WBC: White blood cells
ANC: Absolute neutrophil count
PRBC: Packed red blood cell transfusion
Normal values
31% to 48%
150,000 to 500,000
5,000 to 12,000
Over 1,000
Function Oxygen-carrying cells
Blood-clotting cells
Infection- fighting cells
Infection- fighting cells
Give PRBCs when Hct is less than 20%
Give platelets when child’s PLT is less than 10,000 to 30,000
Date HCT PLT WBC ANC Chemotherapy Other (transfusions, etc.)
Blood Counts
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Recording your child’s blood counts
Lab results recording chart
HCT PLT WBC ANC
Abbreviations:
HCT: Hematocrit
PLT: Platelets
WBC: White blood cells
ANC: Absolute neutrophil count
PRBC: Packed red blood cell transfusion
Normal values
31% to 48%
150,000 to 500,000
5,000 to 12,000
Over 1,000
Function Oxygen-carrying cells
Blood-clotting cells
Infection- fighting cells
Infection- fighting cells
Give PRBCs when Hct is less than 20%
Give platelets when child’s PLT is less than 10,000 to 30,000
Date HCT PLT WBC ANC Chemotherapy Other (transfusions, etc.)
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Caring for Your Child During Gene Therapy
Recording your child’s blood counts
Lab results recording chart
HCT PLT WBC ANC
Abbreviations:
HCT: Hematocrit
PLT: Platelets
WBC: White blood cells
ANC: Absolute neutrophil count
PRBC: Packed red blood cell transfusion
Normal values
31% to 48%
150,000 to 500,000
5,000 to 12,000
Over 1,000
Function Oxygen-carrying cells
Blood-clotting cells
Infection- fighting cells
Infection- fighting cells
Give PRBCs when Hct is less than 20%
Give platelets when child’s PLT is less than 10,000 to 30,000
Date HCT PLT WBC ANC Chemotherapy Other (transfusions, etc.)
Blood Counts
25
Recording your child’s blood counts
Lab results recording chart
HCT PLT WBC ANC
Abbreviations:
HCT: Hematocrit
PLT: Platelets
WBC: White blood cells
ANC: Absolute neutrophil count
PRBC: Packed red blood cell transfusion
Normal values
31% to 48%
150,000 to 500,000
5,000 to 12,000
Over 1,000
Function Oxygen-carrying cells
Blood-clotting cells
Infection- fighting cells
Infection- fighting cells
Give PRBCs when Hct is less than 20%
Give platelets when child’s PLT is less than 10,000 to 30,000
Date HCT PLT WBC ANC Chemotherapy Other (transfusions, etc.)
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Caring for Your Child During Gene Therapy
Resources
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ResourcesThe Immune Deficiency Foundation (IDF)http://primaryimmune.org/ A national non-profit patient organization dedicated to improving the diagnosis, treatment and quality of life of persons with PI through advocacy, education and research. This organization provides information regarding immune mediated disorders.
Washington State Department of Health Parent Page on Newborn Screeninghttp://www.doh.wa.gov/YouandYourFamily/InfantsChildrenandTeens/NewbornScreening/ParentsPage Information regarding diagnosis and newborn screening processes.
Seattle Cancer Care Alliancehttp://www.seattlecca.org/ Brings together the leading research teams and cancer specialists of Fred Hutch, Seattle Children's, and UW Medicine. SCCA partners with Seattle Children's to provide care for immune deficient patients.
SCCA at South Lake UnionThe SCCA outpatient clinic on Lake Union is where most adult patients of Seattle Cancer Care Alliance get care that does not require hospitalization. The clinic also provides transplant related outpatient care to children undergoing bone marrow transplantation.1354 Aloha St. Seattle, WA 98109(855) 557-0555
Directions from freewaysFrom I-5• Take Exit 167, Mercer St./Seattle Center. (If you are northbound on I-5,
you will exit I-5 from the far left lane. If you are southbound, you will exit I-5 from the far right lane.)
• As you exit, move to the far right lane.• Turn right at the end of the ramp onto Fairview Ave. N.• Veer right at the light, staying on Fairview Ave. N. (Do not turn right onto
Valley St.) Travel one block to Aloha St.• Turn right onto Aloha St., and travel two blocks to the SCCA clinic. The
clinic is on your right.• The underground parking garage entrance is just before the clinic circle
driveway, between Yale Ave. N. and Eastlake Ave. E.
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Caring for Your Child During Gene Therapy
From the east via 520• Take 520 west.• Exit onto I-5 south.• Follow the directions above from I-5.
From the east via I-90• Take I-90 west.• Exit onto I-5 north.• Follow the directions above from I-5.
ParkingPatients and their visitors may park in the SCCA clinic underground parking garage. The parking fee is no more than $4 per day. Parking fees are prorated for shorter visits to the clinic. Metered street parking is also available in the area, which is free after 6 p.m., Monday through Saturday, and all day Sunday.
The SCCA and the University of Washington Medical Center Garages (Surgery Pavilion and Triangle) offer same-day reciprocal parking. Bring your receipt from a participating garage to your next appointment that day and it will be honored.
Shuttle and Transit• SCCA’s free shuttle runs between the SCCA outpatient clinic on Lake
Union and UW Medical Center, Seattle Children's, Pete Gross House, and the SCCA House.
• A Metro Bus route near the clinic is the 70. This bus stops on Fairview Avenue N. and runs between downtown Seattle and the University District.
• Seattle Streetcar operates between downtown and the SCCA outpatient clinic on Lake Union.
• Transportation options include volunteer drivers, taxis, and airport shuttles.
Glossary
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Glossary Commonly used terms and their meanings
You will hear many different medical words during your care. Here are some common terms and their definitions.ANC: Absolute neutrophil count. A laboratory procedure which determines the number of neutrophils (a type of white blood cell) in a blood sample.Anticoagulant: An agent that decreases the ability of the blood to clot.Apheresis: The process of removing whole blood from a patient and sending it through a centrifuge, which separates the whole blood into layers of various components.Attending physician: A doctor who leads the healthcare team and supervises the medical care provided to the patient.Autologous Transplant (autograft): A transplant in which the bone marrow or peripheral blood stem cells are taken from the patient, frozen, and later returned to the patient.Bacterial contamination: The presence of bacteria which makes a solution, surface, or body organ impure. For example, bacterial contamination of the blood.Biotechnology: Engineering of procedures and agents used to alter or influence living organisms and processes.Blood draw: The removal of a small sample of blood from a vein. Also called “labs.”Bone marrow: The soft, spongy material in the center of bones that produces all of the blood cells in the body, such as white blood cells, red blood cells, and platelets.Bone marrow aspiration: Obtaining a sample of bone marrow using a needle and syringe. This is done to obtain a sample for laboratory study.Bone marrow harvest: Collecting marrow from the hip bones with a needle and syringe for use in transplant. Marrow is harvested in the operating room.Bone marrow transplant: The process of treating disease with chemotherapy and then giving stem cells (directly from bone marrow, peripheral blood, or cord blood) to restore the production of blood cells.Caregiver: A responsible family member or friend who is able to provide physical care, observational care, and emotional support for a patient in the hospital and home setting.Catheter: A rubber or plastic tube that is inserted into the body to drain fluids or deliver fluids or medication.
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Caring for Your Child During Gene Therapy
Cell separator: A mechanical device used to collect blood cells and separate the various blood components, such as red cells and white cells.Central Intravenous Catheter(CVC)/Line: A small, flexible tube inserted into a large vein near the heart. This serves as a route for medications, fluids, and to obtain blood samples.Central line placement: The process of inserting a catheter into a large vein. This is a minor surgical procedure performed in the operating room under either local or general anesthesia.Chemotherapy: Medications used to destroy or slow growth of abnormal cells, usually cancer cells. In this case, chemotherapy is used to make room for new cells.Collection: The process of accumulating or keeping together, such as collecting stem cells.Complete Blood Count (CBC): A laboratory procedure that determines the number of red blood cells, white blood cells, and platelets in a sample of blood.Complication: An undesired symptom or medical problem that is a consequence of therapy, or another disease.Conditioning: The treatment given to the patient before transplant, intended to kill cells and prepare bone marrow to receive stem cells. Conditioning can be myeloablative (full intensity), in which the recipient’s bone marrow is completely destructed, or non-myeloablative (reduced intensity), in which the recipient’s bone marrow function does return. In this case, conditioning consists of chemotherapy and is non-myeloablative.Counts: Refers to monitoring blood parameters such as ANC and development of lymphocytes and other blood components when watching for engraftment.Donor: A person who gives stem cells to be infused into the patient following chemotherapy. A donor may be the patient, family member, or unrelated person.Erythrocytes: Red blood cells carry oxygen on hemoglobin from the lungs throughout the body.Engraftment: The process by which the normal growth and production of blood cells and the bone marrow resumes.Exit site: The place on the chest where the central intravenous catheter emerges from the skin.FHCRC: Fred Hutchinson Cancer Research Center. The “Hutch” or “Hutchinson Center.”
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Growth factors: Substances naturally occurring in the body that control the production and function of blood cells. These may be given after transplant to speed up engraftment, or given to donors to increase the number of stem cells in the blood stream that will be collected for transplant.Immune system: A complex network of organs, cells, and specialized substances distributed throughout the body which defend it from foreign invaders that cause infection or disease.Immunosuppression: A decrease in the ability of the immune system to fight infection that can be caused by chemotherapy or GVHD therapy.Informed consent: How a patient learns about and understands the purpose, potential risks, and benefits of a research protocol and then agrees to participate. A patient may decline to participate.Infusion: Administration of medications or fluids into a vein over a period of time.Infusion pump: A device used to infuse medications or fluids through a catheter into a vein over a specific amount of time.Intravenous (IV): A route of administration for fluids or medications that are given into a vein.Leukocytes: White blood cells that play a major role in the body’s immune system.Lymphocytes: A type of white blood cell that is part of the immune system. These include B cells and T cells.Mobilization: Stimulating the bone marrow to produce and release large numbers of stem cells into the bloodstream.Mucositis: Reddening and soreness of the tongue, lips, mouth, throat, and gastrointestinal tract as a result of chemotherapy. Mucositis can include painful mouth sores.Neutropenia: Low neutrophil (type of white blood cell) count. A person with neutropenia is at high risk for developing an infection.Neutrophil: A type of white blood cell that provides defense against infection.Peripheral Blood Stem Cells (PBSCs): Cells made in the bone marrow that are circulating in the blood stream and that have the ability to develop into white blood cells, red blood cells, or platelets.Peripheral Blood Stem Cell Transplant: Stem cells circulating in the bloodstream are collected and later given back to a patient after chemotherapy is given.Platelets: Tiny disc-shaped blood cells that help the blood to clot and help prevent bleeding.
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Pneumonia: A disease in which the lungs are inflamed or infected.Protocol: The plan for a specific treatment; may be a scientific study such as a research protocol.Protocol consent forms: A document that outlines the treatment plan/research study that the patient signs to indicate understanding and consent.Red blood cell: A type of blood cell (also called erythrocyte) that carries oxygen in the body.Rejection: A process by which transplanted cells are attacked by the patient’s immune system.SCCA: Seattle Cancer Care Alliance.Side effect: An additional and usually undesirable effect from a drug or other treatment.Sinusoidal Obstruction Syndrome (SOS): A disease caused by obstruction of blood flow through the small blood vessels in the liver, resulting in damage to the liver.Standard treatment: A treatment or other intervention currently being used that has been proven effective on the basis of past clinical studies.Stem cell: An immature cell that is the “parent” cell of the blood cell and has the capacity to mature into a red blood cell, white blood cell, or platelet. The other name for stem cells is CD34+ cells.Tissue typing: Tests that determine how closely the tissues of a donor and the patient match.TPN (total parenteral nutrition): Nutritional support given intravenously. Also called hyperalimentation.Venous access: A method used to gain entrance into a vein.Viable: Capable of living.White blood cells: A group of blood cells, the leukocytes, that helps fight infection.Wiskott-Aldrich syndrome (WAS): A primary immunodeficiency disease in which there is abnormal function of B-lymphocytes and T-lymphocytes, as well as small and dysfunctional platelets.X-linked severe combined immunodeficiency (X-SCID): The most common form of SCID, a potentially fatal syndrome of diverse genetic causes in which there is combined absence of T-lymphocyte and B-lymphocyte function (and in many cases also natural killer, or NK, lymphocyte function).
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Caring for Your Child During Gene Therapy
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Seattle Children’s offers interpreter services for Deaf, hard of hearing or non-English speaking patients, family members and legal representatives free of charge. Seattle Children’s will make this information available in alternate formats upon request. Call the Family Resource Center at 206-987-2201.
This handbook has been reviewed by clinical staff at Seattle Children’s. However, your child’s needs are unique. Before you act or rely upon this information, please talk with your child’s healthcare provider.
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Program for Cell and Gene Therapy
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