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*For medical professionals only
Hematopoietic stem cell transplantation (HSCT) offers the possibility
of curing a variety of benign and malignant hematological diseases.
Benefiting from improved transplant strategies and supportive care, the long-term survival rate of children after transplantation continues to improve, and their quality of life is also receiving increasing attention
.
On October 15, 2022, the "5th Children's Hereditary Diseases and Hematopoietic Stem Cell Transplantation Summit Forum" was held
online.
At the conference, Professor Sun Yuan, President and Director of the Department of Hematology, Beijing Kyoto Children's Hospital, introduced and shared
the relevant content on the topic of "Quality of Life Management after Hematopoietic Stem Cell Transplantation".
This article organizes the clinical experience and academic focus of the lecture topic for the reader.
HSCT began to be used in clinical practice in the 50s of the last century, and after more than 70 years of development, its technology has become increasingly perfect, the types of diseases treated have been expanded, and the cure situation has also shown an upward trend
.
For many benign pathologies, long-term survival rates with HSCT can reach >
90%.
In this case, as the beginning of a life process, the purpose of transplantation is not only to treat the disease and prolong life, but also to ensure a high quality of life
by providing all-round care.
At present, the international quality of life assessment system for diseases, especially chronic diseases, mainly uses health-related quality of life (HRQoL) for evaluation
.
HRQoL is a model for comprehensively evaluating the health status of children from five aspects: physiological state, psychological state, social function state, role function state and overall health state, which can reflect the impact of
health status on the quality of life.
Studies have shown that the effects of childhood age, post-transplantation, parental emotional functioning, and clinical complications will affect HRQoL score results [1].
Another method that can be used to assess quality of life is the Child Quality of Life Measurement Scale, which evaluates and predicts scores based on five aspects: mental, physiological, emotional, social and school functioning, through questionnaires and objective indicators [2].
The scale is a straightforward and effective tool
for assessing and predicting chronic disease management in children.
HSCT is a treatment method in which children receive ultra-large doses of radiotherapy or chemotherapy to remove tumor cells and abnormal clonal cells from the body, and then infuse hematopoietic stem cells collected from themselves or others to rebuild normal hematopoietic and immune function [3].
There are several factors that affect quality of life during HSCT treatment
.
Side effects from pre-treatment of radiotherapy or chemotherapy prior to transplantation are among
the factors that affect quality of life after transplantation.
At present, the main chemotherapy drugs used are alkylating agents, podophyllotoxes and platinum, and the common side effects include: cataracts, pulmonary fibrosis, nephrotoxicity, ototoxicity, cardiovascular toxicity and sexual dysfunction [4].
The main regimens of radiotherapy are systemic radiation therapy (TBI), which has a wider range of side effects and a deeper degree of damage than chemotherapy [4].
Therefore, when pre-treatment with radiation or chemotherapy, it is necessary to assess the benefits and harms and choose a pre-treatment regimen
that resolves the problem and is less risky.
Post-transplant complications can also have an impact on
a child's quality of life.
The most common complications after transplantation include acute and chronic graft-versus-host disease (GVHD), infection, etc.
[4].
Chronic GVHD affects not only the epithelial tissue (gastrointestinal, liver, skin, and lungs) typically affected by acute GVHD, but also any other organ system, including the oral cavity, esophagus, musculoskeletal, joints, fascia, lymphatic hematopoietic system, and genitals [5].
Infections include viruses, bacteria and fungi, and if they cannot be intervened in time, some infections may even cause irreversible damage
to organs.
The long-term effects of HSCT treatment, including endocrine, bone health, sensory, neurocognitive, and secondary tumors, will also have different degrees of impact on children's quality of life [4].
How to better manage the quality of life of children after HSCT treatment? Several studies have provided us with some management ideas
.
The dose and intensity of medication during HSCT treatment, the age at which HSCT is initiated, and lifestyle can all be entry points for quality of life management [6,7].
The experience of the center is to fully combine the actual situation of the child (including the child's age, disease situation, previous treatment, etc.
), comprehensively evaluate the advantages and disadvantages (including the choice of pretreatment, comorbidities, infections, social attributes, etc.
), and check the whole to select the best treatment, transplantation and quality of life management plan
for the child.
It is mainly reflected in the following three aspects:
▌ "subtraction treatment", avoiding unnecessary drugs, reducing the dose
of drugs according to the specific situation of the child, for chemotherapy drugs with large side effects, under the premise of ensuring the efficacy, by choosing alternative drugs or reducing the dose, Reduce its possible adverse consequences
.
Take severe aplastic anemia (SAA) treatment, for example
.
For SAA, the best donor for transplantation is autologous cord blood
.
After autologous cord blood transplantation, the incidence of rejection and GVHD is low, so the use of immunosuppressants is very small or not necessary
.
In pre-transplant pretreatment, the dose can also be reduced to a lower level
.
The use of cyclophosphamide (CTX) is 30-40mg/kg, and the use of flutamide (FLU) and anti-human thymocyte globulin (ATG) is also very small
.
Under this dose of drug treatment, the child's body damage is very small, so it will not have much impact
on the child's quality of life.
In the treatment of SAA's allogeneic transplantation, the focus of the pretreatment process is to avoid affecting
the birth of children by reducing the use of the myeloablative drug busulfane (BU).
Therefore, for children with SAA who have a bone marrow hematopoietic volume of less than 30% and do not have complex long-term transplantation and blood transfusion, BU is not used and the amount
of CTX (100 mg/kg) is reduced.
For children with allogeneic transplantation with bone marrow hematopoietic volume greater than 30% after repeated blood transfusion, very small doses of BU will be selected for treatment
during transplant pretreatment.
▌ "One step ahead", ovarian tissue cryopreservation and transplantation to protect fertility
In the process of HSCT treatment, children with radiotherapy ≥ 5Gy or BU/CTX chemotherapy before bone marrow transplantation have a 20-fold increased risk of early menopause.
The incidence of premature ovarian failure ranges from 70% to 100%.
In addition to reducing fertility, premature ovarian failure can also lead to a decrease in bone density, cognitive impairment, and negative mental and emotional effects [8-10].
Interventions for premature ovarian failure have been proven and effective in cryopreservation and transplantation
of ovarian tissue.
This method has had relatively mature treatment experience and successful cases
in China.
This year, a number of experts in the field also co-authored and published the "Chinese Expert Consensus on the Protection of Fertility in Girls with Hematopoietic Stem Cell Transplantation", which is believed to provide better clinical guidance
for the protection of fertility function of girls with children.
▌ Prevention first, early intervention, multi-means, multi-disciplinary to jointly protect the child's future
Clinically, the selection of donors, the prevention of GVHD by transplantation regimens, and the avoidance of high-risk treatment behaviors for chronic GVHD can be taken as the starting point, and multiple means can be used in conjunction with multiple disciplines for early preventive intervention
.
After a problem, multidisciplinary attention to the recovery of the child's multi-organ function can also effectively ensure the child's quality of
life.
At the same time, the evaluation of transplant indications is also important, through the comprehensive assessment of the child's long-term quality of life and transplant treatment risk, the selection of treatment methods that maximize the benefit of the child is a topic
that clinicians need to pay attention to.
In addition to physical treatment, the psychological comfort and security of children also need to attract the attention
of medical staff, parents and society.
The company of parents, medical staff and the care of the whole society can help children better integrate into society and enhance their sense of
self-identity.
In short, the improvement of the quality of life of children after transplantation treatment is inseparable from the joint efforts
of medical staff, parents and the whole society.
Summary:
The treatment of HSCT is becoming more and more perfect, which brings the possibility
of saving more children.
The quality of life of children after transplantation needs to be paid attention to, and the future of physical and mental health can be created for children while curing diseases
.
Quality of life is related
to many factors before and after the transplantation process.
Curing diseases and thinking deeply about drugs and treatments to reduce and avoid toxic side effects
.
Prevention first, early detection, early intervention, a variety of means, multiple disciplines to jointly protect the future
of the child after transplantation.
Expert profile
Professor Sun Yuan
References: [1] Parsons SK, et al.
Expert Rev Pharmacoecon Outcomes Res.
2013 Apr; 13(2):217-25.
[2]DeCarlo DK,et al.
J AAPOS.
2020 Apr; 24(2):94.
e1-94.
e7.
[3] Yu Zhengzhi, et al.
Journal of Yichun University,2020,42(09):77-80 [4]Chow EJ,et al.
Biol Blood Marrow Transplant.
2016 May; 22(5):782-95.
[5] ZHANG Yu, et al.
International Journal of Blood Transfusion and Hematology,2010(04):315-318 [6]Oberg JA,et al.
Bone Marrow Transplant.
2013 Jun; 48(6):787-93.
[7]Fiuza-Luces C,et al.
Physiology(Bethesda).
2013 Sep; 28(5):330-58.
[8]ISFP Practice Committee,et al.
J Assist Reprod Genet.
2012 Jun; 29(6):465-8.
[9]Letourneau JM,et al.
Nat Rev Clin Oncol.
2011 Jan; 8(1):56-60.
[10]Kim SS,et al.
Fertil Steril.
2011 Apr; 95(5):1535-43.
*This article is only for providing scientific information to medical professionals and does not represent the views of this platform
"We hope that through our efforts, we can make these 'little seedlings' grow into 'towering trees' like ordinary children, become pillars of society, and become pillars of the family
.
"
Hematopoietic stem cell transplantation (HSCT) offers the possibility
of curing a variety of benign and malignant hematological diseases.
Benefiting from improved transplant strategies and supportive care, the long-term survival rate of children after transplantation continues to improve, and their quality of life is also receiving increasing attention
.
On October 15, 2022, the "5th Children's Hereditary Diseases and Hematopoietic Stem Cell Transplantation Summit Forum" was held
online.
At the conference, Professor Sun Yuan, President and Director of the Department of Hematology, Beijing Kyoto Children's Hospital, introduced and shared
the relevant content on the topic of "Quality of Life Management after Hematopoietic Stem Cell Transplantation".
This article organizes the clinical experience and academic focus of the lecture topic for the reader.
HSCT technology is becoming more and more perfect,
A high quality of life has become a new goal of treatment
HSCT began to be used in clinical practice in the 50s of the last century, and after more than 70 years of development, its technology has become increasingly perfect, the types of diseases treated have been expanded, and the cure situation has also shown an upward trend
.
For many benign pathologies, long-term survival rates with HSCT can reach >
90%.
In this case, as the beginning of a life process, the purpose of transplantation is not only to treat the disease and prolong life, but also to ensure a high quality of life
by providing all-round care.
At present, the international quality of life assessment system for diseases, especially chronic diseases, mainly uses health-related quality of life (HRQoL) for evaluation
.
HRQoL is a model for comprehensively evaluating the health status of children from five aspects: physiological state, psychological state, social function state, role function state and overall health state, which can reflect the impact of
health status on the quality of life.
Studies have shown that the effects of childhood age, post-transplantation, parental emotional functioning, and clinical complications will affect HRQoL score results [1].
Another method that can be used to assess quality of life is the Child Quality of Life Measurement Scale, which evaluates and predicts scores based on five aspects: mental, physiological, emotional, social and school functioning, through questionnaires and objective indicators [2].
The scale is a straightforward and effective tool
for assessing and predicting chronic disease management in children.
Review of the course of HSCT treatment,
A variety of factors can affect quality of life
HSCT is a treatment method in which children receive ultra-large doses of radiotherapy or chemotherapy to remove tumor cells and abnormal clonal cells from the body, and then infuse hematopoietic stem cells collected from themselves or others to rebuild normal hematopoietic and immune function [3].
There are several factors that affect quality of life during HSCT treatment
.
Side effects from pre-treatment of radiotherapy or chemotherapy prior to transplantation are among
the factors that affect quality of life after transplantation.
At present, the main chemotherapy drugs used are alkylating agents, podophyllotoxes and platinum, and the common side effects include: cataracts, pulmonary fibrosis, nephrotoxicity, ototoxicity, cardiovascular toxicity and sexual dysfunction [4].
The main regimens of radiotherapy are systemic radiation therapy (TBI), which has a wider range of side effects and a deeper degree of damage than chemotherapy [4].
Therefore, when pre-treatment with radiation or chemotherapy, it is necessary to assess the benefits and harms and choose a pre-treatment regimen
that resolves the problem and is less risky.
Post-transplant complications can also have an impact on
a child's quality of life.
The most common complications after transplantation include acute and chronic graft-versus-host disease (GVHD), infection, etc.
[4].
Chronic GVHD affects not only the epithelial tissue (gastrointestinal, liver, skin, and lungs) typically affected by acute GVHD, but also any other organ system, including the oral cavity, esophagus, musculoskeletal, joints, fascia, lymphatic hematopoietic system, and genitals [5].
Infections include viruses, bacteria and fungi, and if they cannot be intervened in time, some infections may even cause irreversible damage
to organs.
The long-term effects of HSCT treatment, including endocrine, bone health, sensory, neurocognitive, and secondary tumors, will also have different degrees of impact on children's quality of life [4].
Comprehensive assessment, overall check,
Individualized quality of life management benefits more children
How to better manage the quality of life of children after HSCT treatment? Several studies have provided us with some management ideas
.
The dose and intensity of medication during HSCT treatment, the age at which HSCT is initiated, and lifestyle can all be entry points for quality of life management [6,7].
The experience of the center is to fully combine the actual situation of the child (including the child's age, disease situation, previous treatment, etc.
), comprehensively evaluate the advantages and disadvantages (including the choice of pretreatment, comorbidities, infections, social attributes, etc.
), and check the whole to select the best treatment, transplantation and quality of life management plan
for the child.
It is mainly reflected in the following three aspects:
▌ "subtraction treatment", avoiding unnecessary drugs, reducing the dose
of drugs according to the specific situation of the child, for chemotherapy drugs with large side effects, under the premise of ensuring the efficacy, by choosing alternative drugs or reducing the dose, Reduce its possible adverse consequences
.
Take severe aplastic anemia (SAA) treatment, for example
.
For SAA, the best donor for transplantation is autologous cord blood
.
After autologous cord blood transplantation, the incidence of rejection and GVHD is low, so the use of immunosuppressants is very small or not necessary
.
In pre-transplant pretreatment, the dose can also be reduced to a lower level
.
The use of cyclophosphamide (CTX) is 30-40mg/kg, and the use of flutamide (FLU) and anti-human thymocyte globulin (ATG) is also very small
.
Under this dose of drug treatment, the child's body damage is very small, so it will not have much impact
on the child's quality of life.
In the treatment of SAA's allogeneic transplantation, the focus of the pretreatment process is to avoid affecting
the birth of children by reducing the use of the myeloablative drug busulfane (BU).
Therefore, for children with SAA who have a bone marrow hematopoietic volume of less than 30% and do not have complex long-term transplantation and blood transfusion, BU is not used and the amount
of CTX (100 mg/kg) is reduced.
For children with allogeneic transplantation with bone marrow hematopoietic volume greater than 30% after repeated blood transfusion, very small doses of BU will be selected for treatment
during transplant pretreatment.
▌ "One step ahead", ovarian tissue cryopreservation and transplantation to protect fertility
In the process of HSCT treatment, children with radiotherapy ≥ 5Gy or BU/CTX chemotherapy before bone marrow transplantation have a 20-fold increased risk of early menopause.
The incidence of premature ovarian failure ranges from 70% to 100%.
In addition to reducing fertility, premature ovarian failure can also lead to a decrease in bone density, cognitive impairment, and negative mental and emotional effects [8-10].
Interventions for premature ovarian failure have been proven and effective in cryopreservation and transplantation
of ovarian tissue.
This method has had relatively mature treatment experience and successful cases
in China.
This year, a number of experts in the field also co-authored and published the "Chinese Expert Consensus on the Protection of Fertility in Girls with Hematopoietic Stem Cell Transplantation", which is believed to provide better clinical guidance
for the protection of fertility function of girls with children.
▌ Prevention first, early intervention, multi-means, multi-disciplinary to jointly protect the child's future
Clinically, the selection of donors, the prevention of GVHD by transplantation regimens, and the avoidance of high-risk treatment behaviors for chronic GVHD can be taken as the starting point, and multiple means can be used in conjunction with multiple disciplines for early preventive intervention
.
After a problem, multidisciplinary attention to the recovery of the child's multi-organ function can also effectively ensure the child's quality of
life.
At the same time, the evaluation of transplant indications is also important, through the comprehensive assessment of the child's long-term quality of life and transplant treatment risk, the selection of treatment methods that maximize the benefit of the child is a topic
that clinicians need to pay attention to.
In addition to physical treatment, the psychological comfort and security of children also need to attract the attention
of medical staff, parents and society.
The company of parents, medical staff and the care of the whole society can help children better integrate into society and enhance their sense of
self-identity.
In short, the improvement of the quality of life of children after transplantation treatment is inseparable from the joint efforts
of medical staff, parents and the whole society.
Summary:
The treatment of HSCT is becoming more and more perfect, which brings the possibility
of saving more children.
The quality of life of children after transplantation needs to be paid attention to, and the future of physical and mental health can be created for children while curing diseases
.
Quality of life is related
to many factors before and after the transplantation process.
Curing diseases and thinking deeply about drugs and treatments to reduce and avoid toxic side effects
.
Prevention first, early detection, early intervention, a variety of means, multiple disciplines to jointly protect the future
of the child after transplantation.
Expert profile
Professor Sun Yuan
- President / Director of the Department of Hematology and Oncology, Beijing Kyoto Children's Hospital
- Member of the Cellular Society of International Organizations
- Vice President of the Council of the Integrative Oncology Committee of the World Federation of Chinese Medicine Societies
- Vice Chairman of the Child and Adolescent Health Promotion Committee of the Chinese Society for Health Information and Health Big Data
- Vice Chairman of the Cord Blood Application Professional Committee of the China Maternal and Child Health Association
- Member of the Pediatric Blood Diseases and Health Care Group of the Pediatric Diseases and Health Care Branch of the Chinese Maternal and Child Health Association
- Member of the First Committee of Hematology and Oncology Specialty Alliance of China National Children's Medical Center
- Member of the Expert Committee of the Beijing Branch of China Bone Marrow Bank
- Member of the Standing Committee of the First Pediatric Oncology Professional Committee of the Chinese Research Hospital Association
- Director of Pediatric Branch of China Information Research Association of Traditional Chinese Medicine
- Director of China Association of Non-public Medical Institutions
- Vice Chairman of the Pediatric Professional Committee of China Association of Non-public Medical Institutions
- Member of the Standing Committee of Hematopoietic Stem Cell Transplantation Professional Committee of Beijing Society for Cancer Prevention and Control
- Member of Pediatric Oncology Professional Committee of Beijing Anti-Cancer Association
- Director of Pediatric Specialist Branch of Beijing Medical Doctor Association
- Standing Director of Beijing Association of Non-public Medical Institutions
- President of Beijing Changping District Association of Non-public Medical Institutions
- Expert of the investigation and diagnosis team of abnormal reactions to vaccination in Changping District, Beijing
References: [1] Parsons SK, et al.
Expert Rev Pharmacoecon Outcomes Res.
2013 Apr; 13(2):217-25.
[2]DeCarlo DK,et al.
J AAPOS.
2020 Apr; 24(2):94.
e1-94.
e7.
[3] Yu Zhengzhi, et al.
Journal of Yichun University,2020,42(09):77-80 [4]Chow EJ,et al.
Biol Blood Marrow Transplant.
2016 May; 22(5):782-95.
[5] ZHANG Yu, et al.
International Journal of Blood Transfusion and Hematology,2010(04):315-318 [6]Oberg JA,et al.
Bone Marrow Transplant.
2013 Jun; 48(6):787-93.
[7]Fiuza-Luces C,et al.
Physiology(Bethesda).
2013 Sep; 28(5):330-58.
[8]ISFP Practice Committee,et al.
J Assist Reprod Genet.
2012 Jun; 29(6):465-8.
[9]Letourneau JM,et al.
Nat Rev Clin Oncol.
2011 Jan; 8(1):56-60.
[10]Kim SS,et al.
Fertil Steril.
2011 Apr; 95(5):1535-43.
*This article is only for providing scientific information to medical professionals and does not represent the views of this platform
