Bone Marrow Transplantation
Antineoplastic Agents, Alkylating
Hepatic Veno-Occlusive Disease
Graft vs Host Disease
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Combined Modality Therapy
Antineoplastic Combined Chemotherapy Protocols
Leukemia, Myeloid, Acute
Bone marrow transplantation in pediatric patients with therapy-related myelodysplasia and leukemia. (1/594)Eleven children underwent BMT for therapy-related MDS or leukemia, four from HLA-identical siblings and seven from unrelated donors. Ten of the 11 were conditioned with busulfan and cyclophosphamide as the majority had received prior irradiation to the chest and/or abdomen. All patients engrafted. Regimen-related toxicity was more common when compared to historical controls. Eight patients developed acute GVHD and four of eight who survived 100 days post transplant developed extensive chronic GVHD. Non-relapse related mortality occurred in three patients. Five patients developed recurrent malignancy: one died from recurrence of osteosarcoma, three died of recurrent leukemia or MDS and another developed two subsequent malignancies (duodenal carcinoma and anaplastic astrocytoma). Three survive disease-free at 14+, 22+ and 43+ months for a 2 year actuarial cancer-free survival of 24% (95% confidence interval = 5-53%). Although allogeneic BMT can be curative, regimen-related toxicity is frequent and recurrent malignancy remains the major obstacle. (+info)
High dose chemotherapy with busulfan, cyclophosphamide, and etoposide as conditioning regimen for allogeneic bone marrow transplantation for patients with acute myeloid leukemia in first complete remission. (2/594)We explored the combination of busulfan/cyclophosphamide/etoposide as conditioning regimen prior to bone marrow transplantation in 31 patients with acute myeloid leukemia (AML) in first complete remission. The preparative regimen consisted of 16 mg/kg busulfan, 30-60 mg/kg VP-16, and 120 mg/kg cyclophosphamide. With a median follow-up of 30.5 months (range, 5-60 months), 25 patients are alive in continuous complete remission. Estimated disease-free survival at 5 years is 80.5%. Death was due to transplant-related toxicity (graft-versus-host disease and cytomegalovirus infection, graft-versus-host disease and pneumonia, sepsis and mucositis, respectively). None of the patients have relapsed. As demonstrated by the results of this analysis, the conditioning regimen busulfan/cyclophosphamide/etoposide is effective and well tolerated in patients with AML in first complete remission. Main nonhematological toxicities were mucositis and hepatotoxicity. The low mortality and relapse rate appears to justify allogeneic bone marrow transplantation for patients with AML in first complete remission who have an HLA-identical donor. Whether this regimen offers a substantial improvement in disease-free and overall survival over presently used regimens warrants further investigation. (+info)
Busulphan is active against neuroblastoma and medulloblastoma xenografts in athymic mice at clinically achievable plasma drug concentrations. (3/594)High-dose busulphan-containing chemotherapy regimens have shown high response rates in children with relapsed or refractory neuroblastoma, Ewing's sarcoma and medulloblastoma. However, the anti-tumour activity of busulfan as a single agent remains to be defined, and this was evaluated in athymic mice bearing advanced stage subcutaneous paediatric solid tumour xenografts. Because busulphan is highly insoluble in water, the use of several vehicles for enteral and parenteral administration was first investigated in terms of pharmacokinetics and toxicity. The highest bioavailability was obtained with busulphan in DMSO administered i.p. When busulphan was suspended in carboxymethylcellulose and given orally or i.p., the bioavailability was poor. Then, in the therapeutic experiments, busulphan in DMSO was administered i.p. on days 0 and 4. At the maximum tolerated total dose (50 mg kg(-1)), busulphan induced a significant tumour growth delay, ranging from 12 to 34 days in the three neuroblastomas evaluated and in one out of three medulloblastomas. At a dose level above the maximum tolerated dose, busulphan induced complete and partial tumour regressions. Busulphan was inactive in a peripheral primitive neuroectodermal tumour (PNET) xenograft. When busulphan pharmacokinetics in mice and humans were considered, the estimated systemic exposure at the therapeutically active dose in mice (113 microg h ml(-1)) was close to the mean total systemic exposure in children receiving high-dose busulphan (102.4 microg h ml(-1)). In conclusion, busulphan displayed a significant anti-tumour activity in neuroblastoma and medulloblastoma xenografts at plasma drug concentrations which can be achieved clinically in children receiving high-dose busulphan-containing regimens. (+info)
Autologous transplantation of chemotherapy-purged PBSC collections from high-risk leukemia patients: a pilot study. (4/594)We have recently demonstrated that the combination of the alkylating agent nitrogen mustard (NM) and etoposide (VP-16) is capable of eliminating, ex vivo, leukemic cells contaminating PBSC collections and this is associated with a significant recovery of primitive and committed hematopoietic progenitor cells. Based on these data a pilot study on autologous transplantation of NM/VP-16 purged PBSC for high-risk leukemic patients was recently initiated. Twelve patients (seven females and five males) with a median age of 46 years (range 18-57) have been treated. Two patients had acute myeloblastic leukemia (AML) resistant to conventional induction treatment, four patients had secondary AML in I complete remission (CR), one patient was in II CR after failing a previous autologous BM transplantation, while two additional AML individuals were in I CR achieved after three or more cycles of induction treatment. Two patients with high-risk acute lymphoblastic leukemia (ALL) in I CR and one patient with mantle cell lymphoma and leukemic dissemination were also included. Eight patients showed karyotypic abnormalities associated with a poor clinical outcome. The mobilizing regimens included cytosine arabinoside and mitoxantrone with (n = 6) or without fludarabine (n = 3) followed by subcutaneous administration of G-CSF (5 microg/kg/day until the completion of PBSC collection) and G-CSF alone (n = 3) (15 microg/kg/day). A median of two aphereses (range 1-3) allowed the collection of 7.2 x 10(8) TNC/kg (range 3.4-11.5), 5 x 10(6) CD34+ cells/kg (range 2.1-15.3) and 9.2 x 10(4) CFU-GM/kg (0.3-236). PBSC were treated with a constant dose of 20 microg of VP-16/ml and a median individual-adjusted dose (survival < or = 5% of steady-state BM CFU-GM) of NM of 0.7 microg/ml (range 0.25-1.25). Eleven patients were reinfused after busulfan (16 mg/kg) and Cy (120 mg/kg) conditioning with a median residual dose of 0.3 x 10(4) CFU-GM/kg (0-11.5). The median time to neutrophil engraftment (>0.5 x 10(9)/l) for evaluable patients was 25 days (range 12-59); the median time to platelet transfusion independence (>20 and >50 x 10(9)/l) was 40 days (18-95) and 69 days (29-235), respectively. Hospital discharge occurred at a median of 25 days (18-58) after stem cell reinfusion. Four individuals are alive in CR (n = 3) or with residual nodal disease (n = 1 lymphoma patient) with a follow-up of 32, 26, 3 and 14 months, respectively. Seven patients died due to disease progression or relapse (n = 5) or extrahematological transplant toxicity (n = 2). Our data suggest that pharmacological purging of leukapheresis collections of leukemic patients at high-risk of relapse is feasible and ex vivo treated cells reconstitute autologous hematopoiesis. (+info)
Correction of bone marrow failure in dyskeratosis congenita by bone marrow transplantation. (5/594)Dyskeratosis congenita is recognized by its dermal lesions and constitutional aplastic anemia in some cases. We report successful allogeneic bone marrow transplantation in two siblings with this disease from their sister, and their long term follow-up. We used reduced doses of cyclophosphamide and busulfan for conditioning instead of total body irradiation. Also, we report late adverse effects of transplantation which are not distinguishable from the natural course of disease. (+info)
Increased risk of chronic graft-versus-host disease, obstructive bronchiolitis, and alopecia with busulfan versus total body irradiation: long-term results of a randomized trial in allogeneic marrow recipients with leukemia. Nordic Bone Marrow Transplantation Group. (6/594)Leukemic patients receiving marrow from HLA-identical sibling donors were randomized to treatment with either busulfan 16 mg/kg (n = 88) or total body irradiation ([TBI] n = 79) in addition to cyclophosphamide 120 mg/kg. The patients were observed for a period of 5 to 9 years. Busulfan-treated patients had an increased risk of veno-occlusive disease (VOD) of the liver (12% v 1%, P =.01) and hemorrhagic cystitis (32% v 10%, P =.003). Acute graft-versus-host disease (GVHD) was similar in the two groups, but the 7-year cumulative incidence of chronic GVHD was 59% in the busulfan-treated group versus 47% in the TBI group (P =.05). Death from GVHD was more common in the busulfan group (22% v 3%, P <.001). Obstructive bronchiolitis occurred in 26% of the busulfan patients but in only 5% of the TBI patients (P <.01). Complete alopecia developed in 8 busulfan patients and partial alopecia in 17, versus five with partial alopecia in the TBI group (P <.001). Cataracts occurred in 5 busulfan-treated patients and 16 TBI patients (P =.02). The incidence of relapse after 7 years was 29% in both groups. Seven-year transplant-related mortality (TRM) in patients with early disease was 21% in the busulfan group and 12% in the TBI group. In patients with more advanced disease, the corresponding figures were 64% and 22%, respectively (P =.004). Leukemia-free survival (LFS) in patients with early disease was 68% in busulfan-treated patients and 66% in TBI patients. However, 7-year LFS in patients with more advanced disease was 17% in the busulfan group versus 49% in the TBI group (P <.01). In patients with chronic myeloid leukemia (CML) in first chronic phase, 7-year LFS was 72% and 83% in the two groups, respectively. (+info)
Busulphan level and early mortality in thalassaemia patients after BMT. (7/594)The aim of the study was to correlate busulphan (BU) levels of thalassaemia patients with outcome of allogeneic transplant. BU levels were measured by gas chromatography mass fragmentography. All patients received a standardised dose of BU 16 mg/kg, and cyclophosphamide 150 or 200 mg/kg. For area-under-the-curve analysis (AUC), blood samples were obtained at 0, 1, 2, 3, 4 and 6 h after the first and fifth dose for all patients, and additional levels were measured after ninth and/or 13th dose in most patients. Outcome parameters examined included veno-occlusive disease of liver (VOD), idiopathic interstitial pneumonitis, chimerism, and day 90 survival. Twenty consecutive thalassaemia patients who underwent haematopoietic stem cell transplantation were studied. The median age at transplant was 11.2 years (range 3-21 years). Mean BU AUC levels were correlated with age at transplant (r = 0.58, P = 0.007). Nine patients developed VOD and six had mixed chimerism, but these did not correlate with mean BU AUC level. Four patients died before day 50 from VOD and interstitial pneumonitis. Patients with BU AUC levels greater than the median (908 micromol x min/l) had significantly lower probability of survival at day 90 (60%), whereas patients with BU AUC level less than the median all survived beyond day 90. No patient had graft rejection. In conclusion, a high BU AUC level was associated with a higher treatment-related mortality in thalassaemia patients after transplant. (+info)
Combination chemotherapy for choroidal melanoma: ex vivo sensitivity to treosulfan with gemcitabine or cytosine arabinoside. (8/594)Treatment of choroidal melanoma by chemotherapy is usually unsuccessful, with response rates of less than 1% reported for dacarbazine (DTIC)-containing regimens which show 20% or more response rates in skin melanoma. Recently, we reported the activity of several cytotoxic agents against primary choroidal melanoma in an ATP-based tumour chemosensitivity assay (ATP-TCA). In this study, we have used the same method to examine the sensitivity of choroidal melanoma to combinations suggested by our earlier study. Tumour material from 36 enucleated eyes was tested against a battery of single agents and combinations which showed some activity in the previous study. The combination of treosulfan with gemcitabine or cytosine arabinoside showed consistent activity in 70% and 86% of cases, respectively. Paclitaxel was also active, particularly in combination with treosulfan (47%) or mitoxantrone (33%). Addition of paclitaxel to the combination of treosulfan + cytosine analogue added little increased sensitivity. For treosulfan + cytosine arabinoside, further sequence and timing experiments showed that simultaneous administration gave the greatest suppression, with minor loss of inhibition if the cytosine analogue was given 24 h after the treosulfan. Administration of cytosine analogue 24 h before treosulfan produced considerably less inhibition at any concentration. While we have so far been unable to study metastatic tumour from choroidal melanoma patients, the combination of treosulfan with gemcitabine or cytosine arabinoside shows activity ex vivo against primary tumour tissue. Clinical trials are in progress. (+info)
Busulfan is a chemotherapy drug that is used to treat various types of cancer, including leukemia, lymphoma, and multiple myeloma. It works by damaging the DNA of cancer cells, which prevents them from dividing and growing. Busulfan is usually given orally or intravenously, and it can also be used as a conditioning agent before a bone marrow transplant. The drug can cause side effects such as nausea, vomiting, hair loss, and low blood cell counts. It is important to closely monitor patients who are taking busulfan to ensure that the drug is working as intended and to manage any side effects that may occur.
Bone marrow transplantation (BMT) is a medical procedure in which healthy bone marrow is transplanted into a patient who has damaged or diseased bone marrow. The bone marrow is the spongy tissue found inside bones that produces blood cells, including red blood cells, white blood cells, and platelets. There are two main types of bone marrow transplantation: autologous and allogeneic. Autologous BMT involves transplanting bone marrow from the patient's own body, usually after it has been harvested and stored before the patient undergoes high-dose chemotherapy or radiation therapy to destroy their diseased bone marrow. Allogeneic BMT involves transplanting bone marrow from a donor who is a genetic match for the patient. BMT is used to treat a variety of conditions, including leukemia, lymphoma, multiple myeloma, sickle cell anemia, and some inherited blood disorders. The procedure can also be used to treat certain immune system disorders and some genetic diseases. The success of BMT depends on several factors, including the type and stage of the patient's disease, the patient's overall health, and the availability of a suitable donor. The procedure can be complex and may involve several stages, including preparatory treatment, the actual transplantation, and post-transplantation care.
Antineoplastic agents, alkylating are a class of chemotherapy drugs that work by damaging the DNA of cancer cells, which prevents them from dividing and growing. These drugs are called alkylating agents because they contain a group of atoms that can attach to DNA and alter its structure. Alkylating agents are often used to treat a variety of cancers, including leukemia, lymphoma, and ovarian cancer. They can be given by mouth, injection, or infusion, and they may be used alone or in combination with other cancer treatments. However, alkylating agents can also cause side effects, such as nausea, vomiting, hair loss, and an increased risk of infection.
Hepatic Veno-Occlusive Disease (VOD) is a rare but serious condition that affects the liver. It is also known as sinusoidal obstruction syndrome (SOS) or veno-occlusive disease of the liver (VOD/L). VOD occurs when the small blood vessels in the liver (sinusoids) become blocked or narrowed, leading to liver damage and dysfunction. VOD can be caused by a variety of factors, including chemotherapy, radiation therapy, stem cell transplantation, and exposure to certain toxins or medications. The symptoms of VOD can include jaundice, abdominal pain, nausea, vomiting, fatigue, and loss of appetite. In severe cases, VOD can lead to liver failure and death. Treatment for VOD typically involves managing the symptoms and addressing the underlying cause of the condition. This may include medications to reduce inflammation and improve liver function, as well as supportive care to manage symptoms and prevent complications. In some cases, a liver transplant may be necessary to restore liver function.
Cyclophosphamide is an immunosuppressive drug that is commonly used to treat various types of cancer, including lymphoma, leukemia, and multiple myeloma. It works by inhibiting the growth and division of cells, including cancer cells, and by suppressing the immune system. Cyclophosphamide is usually administered intravenously or orally, and its dosage and duration of treatment depend on the type and stage of cancer being treated, as well as the patient's overall health. Side effects of cyclophosphamide can include nausea, vomiting, hair loss, fatigue, and an increased risk of infection. It can also cause damage to the kidneys, bladder, and reproductive organs, and may increase the risk of developing certain types of cancer later in life.
Vidarabine, also known as vidarabine phosphate or ara-A, is an antiviral medication used to treat herpes simplex virus (HSV) infections, including genital herpes and herpes encephalitis. It works by inhibiting the replication of the virus, thereby reducing the severity and duration of symptoms. Vidarabine is typically administered intravenously, either as a single dose or as a series of doses over several days. It is not effective against all types of viruses, and its use is limited to treating HSV infections. Common side effects of vidarabine include nausea, vomiting, headache, and fever. More serious side effects are rare, but may include allergic reactions, liver damage, and bone marrow suppression. Vidarabine is a prescription medication and should only be used under the guidance of a healthcare professional.
Graft-versus-host disease (GVHD) is a condition that can occur after a bone marrow or stem cell transplant. It happens when the transplanted cells (the graft) attack the recipient's (the host) tissues and organs. This can cause a range of symptoms, including skin rash, diarrhea, liver problems, and inflammation of the lungs, gut, and blood vessels. GVHD can be a serious and potentially life-threatening complication of transplantation, but it can also be treated with medications and other therapies.
Alkylating agents are a class of chemotherapy drugs that work by forming covalent bonds with DNA, which leads to the disruption of DNA replication and transcription, ultimately causing cell death. These drugs are commonly used to treat various types of cancer, including leukemia, lymphoma, and solid tumors such as breast, ovarian, and testicular cancer. Alkylating agents can be further classified into two categories: nitrogen mustards and alkyl sulfonates. Nitrogen mustards are the oldest and most widely used alkylating agents, while alkyl sulfonates are newer and have fewer side effects. The mechanism of action of alkylating agents involves the formation of alkyl radicals, which react with the DNA molecule to form covalent bonds. These bonds can cause DNA damage, including cross-linking of DNA strands, which can interfere with DNA replication and transcription. Alkylating agents can also cause damage to other cellular components, such as proteins and lipids. The use of alkylating agents in cancer treatment is associated with significant side effects, including nausea, vomiting, hair loss, and an increased risk of developing secondary cancers. However, the benefits of these drugs in treating cancer often outweigh the risks, and they remain an important part of the cancer treatment arsenal.
Melphalan is a chemotherapy drug that is used to treat various types of cancer, including multiple myeloma, ovarian cancer, and breast cancer. It works by interfering with the production of DNA in cancer cells, which prevents them from dividing and growing. Melphalan is usually given intravenously or orally, and its side effects can include nausea, vomiting, hair loss, fatigue, and an increased risk of infection. It is important to note that Melphalan can be toxic to healthy cells as well, so it is typically used in combination with other medications to minimize side effects and increase its effectiveness.
Thiotepa is a chemotherapy drug that is used to treat certain types of cancer, including ovarian cancer, breast cancer, and lung cancer. It works by interfering with the growth and division of cancer cells, which can slow down or stop the growth of tumors. Thiotepa is usually given intravenously (into a vein) or as a solution that is injected directly into the tumor. It can also be given orally (by mouth) in some cases. Thiotepa can cause side effects, including nausea, vomiting, hair loss, and a low white blood cell count. It is important to follow your doctor's instructions carefully when taking thiotepa, as it can be toxic if not used properly.
Hematologic neoplasms are a group of disorders that affect the blood and bone marrow, including the production of blood cells. These disorders are characterized by the abnormal growth and proliferation of blood cells, which can lead to an overproduction of certain types of blood cells (such as leukemias) or a deficiency of certain types of blood cells (such as anemia). Hematologic neoplasms can be either benign (non-cancerous) or malignant (cancerous), and they can affect people of all ages. Some common types of hematologic neoplasms include leukemia, lymphoma, multiple myeloma, and myelodysplastic syndromes. Treatment for hematologic neoplasms typically involves a combination of chemotherapy, radiation therapy, and/or stem cell transplantation.
Leukemia is a type of cancer that affects the blood and bone marrow. It is characterized by the abnormal production of white blood cells, which can interfere with the normal functioning of the immune system and other parts of the body. There are several different types of leukemia, including acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), chronic lymphocytic leukemia (CLL), and chronic myeloid leukemia (CML). Treatment for leukemia typically involves chemotherapy, radiation therapy, and/or stem cell transplantation.
Sulfonic acids are a class of organic compounds that contain a sulfonic acid group (-SO3H) attached to a carbon atom. They are typically colorless or yellowish liquids with a strong, unpleasant odor. In the medical field, sulfonic acids are used as intermediates in the synthesis of various drugs and other chemical compounds. They are also used as disinfectants and as components of some antiseptic solutions. One specific sulfonic acid that is commonly used in medicine is sodium sulfacetamide, which is used to treat acne and other skin infections. It works by inhibiting the growth of bacteria on the skin. Other sulfonic acids, such as sodium sulfite, are used as preservatives in some medications to prevent the growth of bacteria and other microorganisms.
Leukemia, Myelogenous, Chronic, BCR-ABL Positive is a type of cancer that affects the bone marrow and blood cells. It is also known as Chronic Myeloid Leukemia (CML) and is characterized by the presence of an abnormal Philadelphia chromosome, which is caused by a genetic mutation. This mutation results in the production of an abnormal protein called BCR-ABL, which promotes the uncontrolled growth and division of white blood cells. CML is typically diagnosed in adults and is treatable with medications that target the BCR-ABL protein. However, it is a chronic condition that requires lifelong treatment and monitoring.
Myelodysplastic syndromes (MDS) are a group of blood disorders that affect the bone marrow, which is the spongy tissue inside bones where blood cells are produced. In MDS, the bone marrow produces abnormal blood cells that do not function properly, leading to a decrease in the number of healthy blood cells in the body. MDS can cause a range of symptoms, including fatigue, weakness, shortness of breath, and an increased risk of infections and bleeding. The severity of MDS can vary widely, and some people with the condition may not experience any symptoms at all. There are several different types of MDS, which are classified based on the specific characteristics of the abnormal blood cells and the severity of the disease. Treatment for MDS depends on the type and severity of the condition, and may include medications, blood transfusions, or bone marrow transplantation.
Combined modality therapy (CMT) is a cancer treatment approach that involves using two or more different types of treatments simultaneously or in sequence to achieve a better therapeutic effect than any single treatment alone. The goal of CMT is to increase the effectiveness of cancer treatment while minimizing side effects. The different types of treatments that may be used in CMT include surgery, radiation therapy, chemotherapy, immunotherapy, targeted therapy, and hormonal therapy. The specific combination of treatments used in CMT depends on the type and stage of cancer, as well as the patient's overall health and individual needs. CMT is often used for the treatment of advanced or aggressive cancers, where a single treatment may not be effective. By combining different treatments, CMT can help to destroy cancer cells more completely and prevent the cancer from returning. However, CMT can also have more significant side effects than a single treatment, so it is important for patients to discuss the potential risks and benefits with their healthcare provider before starting treatment.
Antilymphocyte serum (ALS) is a type of serum that contains antibodies against lymphocytes, which are a type of white blood cell that plays a crucial role in the immune system. ALS is used in medical treatments to suppress the immune system, particularly in cases where the immune system is overactive or attacking healthy cells. ALS is typically used in the treatment of autoimmune diseases, such as rheumatoid arthritis, lupus, and multiple sclerosis, where the immune system mistakenly attacks the body's own tissues. It is also used in the treatment of certain types of cancer, such as leukemia and lymphoma, where the immune system is weakened and unable to fight off the cancer cells. ALS is prepared by injecting a small amount of lymphocytes into a horse, which then produces antibodies against the lymphocytes. These antibodies are then harvested from the horse's blood and purified to create ALS. The resulting serum contains high levels of antibodies that can bind to and neutralize lymphocytes, thereby suppressing the immune system.
Antineoplastic Combined Chemotherapy Protocols (ACCP) are a type of chemotherapy treatment used to treat cancer. They involve the use of multiple drugs in combination to target and destroy cancer cells. The drugs used in an ACCP are chosen based on the type and stage of cancer being treated, as well as the patient's overall health. The goal of an ACCP is to shrink the tumor, slow the growth of cancer cells, and improve the patient's quality of life.
Leukemia, Myeloid is a type of cancer that affects the myeloid cells in the bone marrow. Myeloid cells are a type of white blood cell that helps fight infections and diseases in the body. In leukemia, myeloid cells grow and divide uncontrollably, leading to an overproduction of these cells in the bone marrow and bloodstream. There are several subtypes of myeloid leukemia, including acute myeloid leukemia (AML) and chronic myeloid leukemia (CML). AML is a rapidly progressing cancer that usually affects older adults, while CML is a slower-growing cancer that is more common in middle-aged and older adults. Symptoms of myeloid leukemia may include fatigue, weakness, fever, night sweats, weight loss, and easy bruising or bleeding. Treatment for myeloid leukemia typically involves chemotherapy, radiation therapy, targeted therapy, and bone marrow transplantation. The prognosis for myeloid leukemia depends on the subtype, age of the patient, and the stage of the disease at diagnosis.
Acute Myeloid Leukemia (AML) is a type of cancer that affects the bone marrow and blood cells. It is characterized by the rapid growth of abnormal white blood cells, called myeloid cells, in the bone marrow. These abnormal cells do not function properly and can crowd out healthy blood cells, leading to a variety of symptoms such as fatigue, weakness, and frequent infections. AML can occur in people of all ages, but it is most common in adults over the age of 60. Treatment for AML typically involves chemotherapy, radiation therapy, and/or stem cell transplantation.
California Institute for Regenerative Medicine
Severe combined immunodeficiency
Bcr-Abl tyrosine-kinase inhibitor
Alkylating antineoplastic agent
Total body irradiation
Hematopoietic stem cell transplantation
Spermatogonial stem cell
Specialty drugs in the United States
ATC code L01
Busulfan - Wikipedia
Busulfan: MedlinePlus Drug Information
1178 APC Code Busulfan injection
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- Busulfan is used in pediatrics and adults in combination with cyclophosphamide or fludarabine/clofarabine as a conditioning agent prior to bone marrow transplantation, especially in chronic myelogenous leukemia (CML) and other leukemias, lymphomas, and myeloproliferative disorders. (wikipedia.org)
- As an adjunct therapy with cyclophosphamide for conditioning prior to bone marrow transplantation in adults and children >12 kg, intravenous (IV) busulfan (Bulsulfex) is dosed at 0.8 mg/kg every six hours for 16 doses (four days). (wikipedia.org)
- Myleran is supplied in white film coated tablets with 2 mg of busulfan per tablet. (wikipedia.org)
- Both IV and oral formulations require prophylactic antiemetic agents administered prior to the busulfan dose and scheduled antiemetics administered thereafter. (wikipedia.org)
- Busulfan therapeutic drug monitoring is completed based on trough (pre-dose) levels with a target six-hour area under the curve (AUC) of between 900 and 1500 micromolxmin. (wikipedia.org)
- However, clinical studies of busulfan were completed with patients taking phenytoin, so no empiric dose adjustment is necessary if patients are taking phenytoin with busulfan. (wikipedia.org)
- Your doctor may need to change your dose or tell you to stop taking busulfan for a period of time to allow your blood count to return to normal if it has dropped too low. (medlineplus.gov)
- Your doctor may adjust your dose of busulfan depending on your response to treatment and any side effects that you experience. (medlineplus.gov)
- Therapeutic drug monitoring (TDM) of Busulfan is essential for management of Bone Marrow Transplant (BMT) patients, by optimizing dose, supporting compliance, and minimizing toxicity. (labroots.com)
- Individualized busulfan dosing improves outcomes compared to fixed-dose administration in pre-transplant minimal residual disease-positive acute myeloid leukemia patients with intermediate-risk undergoing allogeneic stem cell transplantation in CR. (astct.org)
- We hypothesized that a fractionated myeloablative busulfan dose delivered over an extended period would reduce nonrelapse mortality (NRM) while retaining antileukemic effects. (bvsalud.org)
- Toxicity may include interstitial pulmonary fibrosis ("busulfan lung"), hyperpigmentation, seizures, hepatic (veno-occlusive disease) (VOD) or sinusoidal obstruction syndrome (SOS), emesis, and wasting syndrome. (wikipedia.org)
- Busulfan is a chemotherapeutic agent used as a component in a myeloablative preconditioning regimen before hematological stem cell transplantation. (labroots.com)
- Children receive busulfan orally as part of myeloablative therapy before bone marrow transplantation for malignant and nonmalignant conditions. (johnshopkins.edu)
- Myeloablative fractionated busulfan for allogeneic stem cell transplant in older patients or patients with comorbidities. (bvsalud.org)
- Busulfan tablets are also used in combination with other drugs to destroy the bone marrow and cancer cells in preparation for a bone marrow transplant. (medlineplus.gov)
- tell your doctor and pharmacist if you are allergic to busulfan, any other medications, or any of the ingredients in busulfan tablets. (medlineplus.gov)
- Busulfan is an alkylsulfonate. (wikipedia.org)
- Busulfan was the mainstay of the chemotherapeutic treatment of chronic myeloid leukemia (CML) until it was displaced by the new gold standard, imatinib, though it is still in use to a degree as a result of the drug's relative low cost. (wikipedia.org)
- After 2002, a great interest has appeared for intravenous presentations of busulfan. (wikipedia.org)
- Individualized, pharmacokinetic (PK)-guided dosing of busulfan improved relapse rates, leukemia-free survival (LFS), and overall survival (OS) following allogeneic stem cell transplantation (allo-SCT), compared with fixed dosing, in patients with intermediate-risk acute myeloid leukemia who were in complete remission (CR) at the time of transplant. (astct.org)
- To optimize busulfan exposure, dosing was adjusted to achieve an area under the curve (AUC) within ±10% of the target (40, 60, or 89 mg*h/l). (astct.org)
- After a median follow-up of 27 months, 3-year relapses, LFS, and OS were significantly lower after individualized compared with fixed busulfan exposure. (astct.org)
- Busulfan comes as a tablet to take by mouth once a day. (medlineplus.gov)
- Busulfan was approved by the US Food and Drug Administration (FDA) for treatment of chronic myeloid leukemia (CML) in 1999. (wikipedia.org)
- Busulfan clearance rate normalized to surface area is twice as high in children (200 ± 100 mL/min/m 2 ) as it is in adults (95 ± 54 mL/min/m 2 ). (johnshopkins.edu)
- Phenytoin increases hepatic clearance of busulfan (resulting in decreased busulfan AUC). (wikipedia.org)
- How the Busulfan testing improved the management of Bone Marrow Transplant Patients? (labroots.com)
- The TDM and timely plasma concentration reporting of Busulfan would provide an improvement in the quality-of-care for BMT patients. (labroots.com)
- Identify our purpose to measure Busulfan in BMT patients' samples. (labroots.com)
- LFS was significantly improved in patients who were MRD positive by flow cytometry prior to transplant, but not those who were MRD negative, after individualized busulfan administration. (astct.org)
- 32 patients (37%) received PK-guided busulfan dosing. (astct.org)
- The study is the first to analyze the role of individualized busulfan administration in a homogeneous population of patients with intermediate-risk AML in CR prior to allo-SCT, write co-corresponding authors Nicolaus Kröger and Evgeny Klyuchnikov and colleagues at the University of Hamburg in Germany. (astct.org)
- A randomized prospective study to evaluate impact of individualized [busulfan] administration on relapses and survival outcomes in larger cohort of patients focusing on MRD studies is warranted," they conclude. (astct.org)
- The chronic phase varies in duration, depending on the maintenance therapy used: it usually lasts 2-3 years with hydroxyurea (Hydrea) or busulfan therapy, but it may last for longer than 9.5 years in patients who respond well to interferon-alfa therapy. (medscape.com)
- Busulfan can cause a severe decrease in the number of blood cells in your bone marrow. (medlineplus.gov)
- Seizures and VOD are serious concerns with busulfan therapy and prophylaxis is often utilized to avoid these effects. (wikipedia.org)
- Concomitant use of acetaminophen within 72 hours of busulfan use can reduce busulfan clearance (resulting in increased busulfan AUC), as acetaminophen is also metabolized via glutathione and may deplete stores. (wikipedia.org)
- Taking busulfan on an empty stomach is recommended to reduce the risk of nausea and emesis. (wikipedia.org)
- If you take busulfan with other medications that may cause a low blood count, the side effects of the medications may be more severe. (medlineplus.gov)
- A liquid chromatography-tandem mass spectrometry method for rapid and accurate quantification of Busulfan in plasma was developed and validated in the Department of Chemistry at Baptist Hospital of Miami. (labroots.com)
- Describe the validation results of the method to measure Busulfan in plasma by LC-MS/MS. (labroots.com)
- We evaluated the disposition of busulfan in children between 2 months and 3.6 years of age with lysosomal storage diseases, leukemia, and immunodeficiency disorders receiving oral doses of 1 or 2 mg/kg using a gas chromatographic assay. (johnshopkins.edu)
- Participants received busulfan 80 mg/m2 as outpatients on days -20 and -13 before transplant . (bvsalud.org)
- Busulfan also induces impotence in males (kills germ cells), thrombocytopenia, a condition of lowered blood platelet count and activity, and sometimes medullary aplasia. (wikipedia.org)
- Your doctor will order laboratory tests before, during and after your treatment to check your body's response to busulfan to see if your blood cells are affected by this drug. (medlineplus.gov)
- Busulfan is in a class of medications called alkylating agents. (medlineplus.gov)
- Many other medications may also interact with busulfan, so be sure to tell your doctor about all the medications you are taking, even those that do not appear on this list. (medlineplus.gov)
- you should know that busulfan may interfere with the normal menstrual cycle (period) in women, may stop sperm production in men. (medlineplus.gov)