OBJECTIVE: To investigate the efficacy, prognosis and safety of decitabine combined with modified EIAG regimen in the treatment of patients with relapsed/refractory acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS). METHODS: The clinical data of 44 patients with relapsed/refractory AML and high-risk MDS admitted to our hospital from January 2017 to December 2020 were analyzed retrospectively. The patients were equally divided into D-EIAG group (decitabine combined with EIAG regimen) and D-CAG group (decitabine combined with CAG regimen) according to clinical treatment regimen. The complete response (CR), CR with incomplete hematologic recover (CRi), morphologic leukemia-free state (MLFS), partial response (PR), overall response rate (ORR), modified composite complete response (mCRc), overall survival (OS) time, 1-year OS rate, myelosuppression and adverse reactions between the two groups were compared. RESULTS: In D-EIAG group, 16 patients (72.7%) achieved mCRc (CR+CRi+MLFS), 3 patients (13.6%) achieved PR, and ORR (mCRc+PR) was 86.4%. In D-CAG group, 9 patients (40.9%) achieved mCRc, 6 patients (27.3%) achieved PR, and ORR was 68.2%. Difference was observed in mCRc rate between the two groups (P=0.035), but not in ORR (P>0.05). The median OS time of D-EIAG group and D-CAG group was 20 (2-38) months and 16 (3-32) months, and 1-year OS rate was 72.7% and 59.1%, respectively. There was no significant difference in 1-year OS rate between the two groups (P>0.05). After induction chemotherapy, the median time for absolute neutrophil count recovery to 0.5×10⁹/L in D-EIAG group and D-CAG group was 14 (10-27) d and 12 (10-26) d, for platelet count recovery to 20×10⁹/L was 15 (11-28) d and 14 (11-24)d, the median red blood cell suspension transfusion volume was 8 (6-12) U and 6 (6-12) U, and the median apheresis platelet transfusion volume was 4 (2-8) U and 3 (2-6) U, respectively. There were no statistically significant differences in comparison of the above indicators between the two groups (P>0.05). The hematological adverse reactions of patients were mainly myelosuppression. Grade III-IV hematological adverse events occurred in both groups (100%), with no increase in the incidence of non-hematological toxicities such as gastrointestinal reactions or liver function damage. CONCLUSION Decitabine combined with EIAG regimen in the treatment of relapsed/refractory AML and high-risk MDS can improve remission rate, provide an opportunity for subsequent therapies, and have no increase in adverse reactions compared with D-CAG regimen.
Humans ; Decitabine/therapeutic use* ; Treatment Outcome ; Retrospective Studies ; Cytarabine ; Myelodysplastic Syndromes/drug therapy* ; Leukemia, Myeloid, Acute/drug therapy* ; Bone Marrow Diseases/drug therapy* ; Antineoplastic Combined Chemotherapy Protocols/therapeutic use*

OBJECTIVE: To investigate the effect of course delay of CCLG-ALL-2008 regimen on the relapse of paediatric B-cell acute lymphoblastic leukemia (B-ALL) patients. METHODS: Paediatric B-ALL patients newly diagnosed and treated with CCLG-ALL-2008 regimen in the Children's Hospital of Soochow University from January 2011 to December 2014 were retrospectively analyzed to clarify the relationship between chemotherapy course delay and relapse, and explore the causes of course delay which led to relapse. Patients were followed up until July 2019. RESULTS: The correlation between treatment delay (number of weeks) and relapse rate was statistically significant (P=0.034), and hazard ratio indicated that longer than 4 weeks had a significant effect. The effect of positive minimal residual disease (MRD) (1×10^-4≤MRD≤1×10^-2) at the 12th week on the relapse rate was also statistically significant (P=0.041). Among the causes of treatment delay, the effect of myelosuppression on the relapse rate was statistically significant (P=0.01). CONCLUSION Treatment delay exceeding 4 weeks, positive MRD at the 12th week, and myelosuppression are independent prognostic factors for relapse.
Antineoplastic Combined Chemotherapy Protocols/therapeutic use* ; Bone Marrow Diseases/drug therapy* ; Burkitt Lymphoma/drug therapy* ; Child ; Disease-Free Survival ; Humans ; Neoplasm, Residual/drug therapy* ; Precursor Cell Lymphoblastic Leukemia-Lymphoma/drug therapy* ; Prognosis ; Recurrence ; Retrospective Studies ; Treatment Outcome

BACKGROUND/AIMS: Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematologic malignancy that typically presents in the form of skin manifestations with or without lymph node and bone marrow involvement. Given its rarity and recent recognition as a distinct pathological entity, no standard of treatment exists for this aggressive disease and its prognosis is particularly dismal. METHODS: We retrospectively analyzed clinical features and treatment outcomes of patients who were diagnosed with BPDCN between 2000 and 2014. RESULTS: Ten patients had a median age at diagnosis of 41 years (range, 18 to 79), and seven patients were male. Sites of disease involvement were the skin (n = 7), lymph node (n = 5), bone marrow (n = 2), liver (n = 2), spleen (n = 2), and soft tissue (n = 1). Intensified chemotherapy regimens such as hyperCVAD regimen (cyclophosphamide, vincristine, doxorubicin, dexamethasone, methotrexate, cytarabine), and VPDL (vincristine, methylprednisolone, daunorubicin, L-asparaginase) were used as a first-line treatment. Although all patients treated with intensified chemotherapy showed an objective response (five patients with complete response) with median progression-free survival of 11.2 months (range 6.2 to 19.4), complete remission was not sustained for more than 2 years in any case. The response was relatively long-lived compared with previously reported CHOP (doxorubicin, cyclophosphamide, vincristine, prednisone)-like regimens, but the above regimens do not result in long-term remission. CONCLUSIONS: All patients treated with hyperCVAD or VPDL showed an objective response, but the duration of response was relatively short. Thus, the development of more effective induction as well as consolidation treatment strategy should be warranted to improve this rare disease entity.
Bone Marrow ; Cyclophosphamide ; Daunorubicin ; Dendritic Cells* ; Dexamethasone ; Diagnosis ; Disease-Free Survival ; Doxorubicin ; Drug Therapy ; Hematologic Neoplasms ; Humans ; Korea* ; Liver ; Lymph Nodes ; Male ; Methotrexate ; Methylprednisolone ; Prognosis ; Rare Diseases ; Retrospective Studies ; Skin ; Skin Manifestations ; Spleen ; Vincristine

As extra-mammary Paget's disease is rare and usually diagnosed at early stage when it is highly curable with surgical resection, it is much rarer to see patients with recurrent metastatic disease. Thrombotic thrombocytopenic purpura in patients with metastatic solid cancer is also a rare disease and may result from bone marrow metastasis or bone marrow necrosis. For the latter, the majority of cases are not eligible for systemic chemotherapy for rapid disease progression and poor performance status. Herein, authors report a patient with thrombotic thrombocytopenic purpura associated with bone marrow necrosis complicating extra-mammary Paget's disease who was successfully treated with docetaxel and carboplatin combination chemotherapy.
Bone Marrow* ; Carboplatin ; Disease Progression ; Drug Therapy ; Drug Therapy, Combination ; Humans ; Necrosis* ; Neoplasm Metastasis ; Paget Disease, Extramammary* ; Purpura, Thrombotic Thrombocytopenic* ; Rare Diseases

Numerous disorders and etiologies may underlie increased eosinophil counts. Hypereosinophilia (HE) is defined as a peripheral blood eosinophil count greater than 1,500/mm3 and may be potentially harmful because of tissue damage. Hypereosinophilic syndrome (HES) also represents a heterogeneous disorder characterized by persistent HE with the evidence of organ dysfunction, clinical symptoms, or both caused by eosinophilia. The refining criteria and subclassification of HE and HES are currently being revised on cellular and molecular based diagnostic methods. Initial approaches focus on evaluating various underlying causes, including helminthic infections, adverse drug reactions, allergic diseases, and neoplastic diseases. When secondary causes of HE are excluded, the workup should proceed to the evaluation of primary/clonal bone marrow disease, including fip 1-like 1-platelet driven growth factor receptor alpha (FIP1L1-PDGFRA) mutation. Concurrently, if the patient has symptoms and signs, organ damage or dysfunction must be evaluated. Although, corticosteroids are the mainstay of therapy in confirmed HES, imatinib is considered a definitive treatment for FIP1L1-PDGFRA, platelet driven growth factor receptor beta rearranged HE and HES. In this article, we discuss recent advances in the classification of and practical approaches to HE and HES. In addition, we introduce several promising therapies for HE and HES.
Adrenal Cortex Hormones ; Blood Platelets ; Bone Marrow Diseases ; Classification* ; Drug-Related Side Effects and Adverse Reactions ; Eosinophilia ; Eosinophils ; Helminths ; Humans ; Hypereosinophilic Syndrome ; Molecular Targeted Therapy ; Imatinib Mesylate

Hematopoietic stem cell transplantation (HSCT) is the first field where human stem cell therapy was successful. Flooding interest on human stem cell therapy to cure previously incurable diseases is largely indebted to HSCT success. Allogeneic HSCT has been an important modality to cure various diseases including hematologic malignancies, various non-malignant hematologic diseases, primary immunodeficiency diseases, and inborn errors of metabolism, while autologous HSCT is generally performed to rescue bone marrow aplasia following high-dose chemotherapy for solid tumors or multiple myeloma. Recently, HSCs are also spotlighted in the field of regenerative medicine for the amelioration of symptoms caused by neurodegenerative diseases, heart diseases, and others. Although the demand for HSCs has been growing, their supply often fails to meet the demand of the patients needing transplant due to a lack of histocompatible donors or a limited cell number. This review focuses on the generation and large-scale expansion of HSCs, which might overcome current limitations in the application of HSCs for clinical use. Furthermore, current proof of concept to replenish hematological homeostasis from non-hematological origin will be covered.
Bone Marrow ; Cell Count ; Drug Therapy ; Heart Diseases ; Hematologic Diseases ; Hematologic Neoplasms ; Hematopoietic Stem Cell Transplantation ; Hematopoietic Stem Cells* ; Homeostasis ; Humans ; Metabolism, Inborn Errors ; Multiple Myeloma ; Neurodegenerative Diseases ; Regenerative Medicine ; Stem Cells ; Tissue Donors

Persistent bone marrow aplasia after intensive chemotherapy is uncommon, but is one of the fatal complications in patients with acute myeloid leukemia (AML). Although allogeneic hematopoietic stem cell transplantation (HSCT) is considered to be contraindicated for patients who have hematologic diseases with serious infections, such as bacterial septicemia or invasive fungal diseases, combined with prolonged neutropenia due to frequent morbidity and mortality, such risks can be overcome by non-myeloablative conditioning and best supportive care. Here, we report an AML patient with persistent marrow aplasia after induction therapy, treated successfully with reduced-intensity allogeneic HSCT despite severe bacterial and fungal infections.
Anemia, Aplastic ; Bone Marrow* ; Drug Therapy* ; Hematologic Diseases ; Hematopoietic Stem Cell Transplantation ; Humans ; Leukemia, Myeloid, Acute* ; Mortality ; Neutropenia ; Sepsis ; Stem Cell Transplantation* ; Stem Cells*

Behcet's disease is an inflammatory disorder characterized by recurrent oral aphthous ulcers, genital ulcers, uveitis, and skin lesions. A few cases of hematologic disease in patients with Behcet's disease have been reported in the literature. However, acute precursor T cell lymphoblastic leukemia has never been described in association with Behcet's disease. We recently encountered a case of acute precursor T cell lymphoblastic leukemia in a 62-year-old man with a prior diagnosis of Behcet's disease. The patient presented with febrile neutropenia and his bone marrow biopsy revealed acute precursor T cell lymphoblastic leukemia. He was scheduled to undergo therapeutic chemotherapy, but unfortunately he died from pneumonia prior to treatment.
Biopsy ; Bone Marrow ; Diagnosis ; Drug Therapy ; Febrile Neutropenia ; Hematologic Diseases ; Humans ; Middle Aged ; Pneumonia ; Precursor T-Cell Lymphoblastic Leukemia-Lymphoma* ; Skin ; Stomatitis, Aphthous ; Ulcer ; Uveitis

Intravascular large B-cell lymphoma (IVLBCL) is a rare subtype of non-Hodgkin lymphoma. It usually presents with nonspecific symptoms, such as fever, rather than with overt lymphadenopathy. Reports of hypercalcemia, as the initial presentation of IVLBCL, are limited in the literature, despite it being a well-known complication of various solid cancers. We present a 68-year-old male with severe hypercalcemia and increased levels of serum parathyroid hormone-related protein. He was diagnosed with IVLBCL, involving the bone marrow and spleen, and was successfully treated with rituximab-containing chemotherapy. A few previous case reports have shown hypercalcemia in patients with IVLBCL. Much like our case, previous cases with hypercalcemia had advanced diseases, including bone marrow invasion. Although it was an extremely rare manifestation of IVLBCL, we suggest that IVLBCL should be a part of the differential diagnosis in patients with unexplained hypercalcemia. Therefore, an active work-up might be recommended, including positron emission tomography/computed tomography scan and bone marrow examination, which may be useful for early diagnosis.
Aged ; Bone Marrow ; Bone Marrow Examination ; Diagnosis, Differential ; Drug Therapy ; Early Diagnosis ; Electrons ; Fever ; Humans ; Hypercalcemia* ; Lymphatic Diseases ; Lymphoma, B-Cell* ; Lymphoma, Non-Hodgkin ; Male ; Parathyroid Hormone-Related Protein* ; Spleen

OBJECTIVETo observe the clinical effect of Guilu Erxian Glue Cataplasm (GEGC) on carcinoma of the large intestine patients with myelosuppression after chemotherapy, and further to confirm its efficiency and safety.

METHODSTotally 60 patients with carcinoma of the large intestine were randomly assigned to two groups. Meanwhile, they all accepted FOLFIRI chemotherapy. Patients in the treatment group were additionally applied at Shenque (RN8), exchanging once per every other day, for 14 successive days. Patients in the control group took placebos with the same dose and dosage as the treatment group. The blood cell counts (WBC, NE, and PLT) were detected before chemotherapy, at day 7, 10, and 14. The TCM symptoms integrals, Karnofsky performance score (KPS), liver and kidney functions were observed before chemotherapy, at day 7 and day 14. Adverse skin reactions were observed each day. And the usage of hematopoietic growth factors was recorded.

RESULTS(1) The KPS score at day 7 was more stable in the treatment group than in the control group; the WBC and NE counts in the peripheral blood at day 14 were higher in the treatment group than in the control group; and TCM symptoms integrals at day 14 was lower in the treatment group than in the control group, all with statistical difference (P < 0.05). (2) Compared with the control group, the PLT count was higher in the treatment group than in the control group, the usage of rhG-CSF and antibiotics was less in the treatment group than in the control group, all with no statistical difference (P > 0.05). (3) No obvious adverse reactions such as liver injury, renal injury, or skin allergy were observed.

CONCLUSIONSAdjuvant treatment of GEGC could improve carcinoma of the large intestine patients with myelosuppression to some extent. No relevant adverse reactions were found.

Adjuvants, Immunologic ; therapeutic use ; Adult ; Aged ; Bone Marrow Diseases ; chemically induced ; drug therapy ; Colorectal Neoplasms ; drug therapy ; Drugs, Chinese Herbal ; therapeutic use ; Female ; Humans ; Male ; Middle Aged