[Efficacy of different regimens and prognostic factors in patients with first relapsed multiple myeloma treated after front-line bortezomib, cyclophosphamide, and dexamethasone].

Objective: To analyze the efficacy of second-line regimens and prognostic factors in patients with first-relapsed multiple myeloma (MM) treated with bortezomib, cyclophosphamide, and dexamethasone (BCD). Methods: A retrospective cohort study. Clinical data were collected in first-relapsed MM patients after BCD treatment from three tertiary hospitals in north China from July 2009 to October 2022. Patients were classified according to the second-line regimen into the immunotherapy group, single novel agent group [either proteasome inhibitor (PI) or immunomodulatory drug (IMiD)], combination treatment group (both PI+IMiD), and traditional treatment group. Responses to second-line regimens and survival data were analyzed. The Kaplan-Meier method was used for survival analysis and the Cox proportional risk model was used for univariate and multivariate analyses. Results: A total of 217 patients were enrolled including 8.8% (19/217) in the immunotherapy group, 48.4% (105/217) in the PI/IMiD group, 29.9% (65/217) in the PI+IMiD group, and 12.9% (28/217) in the traditional treatment group. The median age was 62 years (range 31-83 years) and 56.2% (122/217) were males. The overall response rates (ORRs) in the four groups were 94.7% (18/19) vs. 56.2% (59/105) vs. 73.8% (48/65) vs. 32.1% (9/28) (χ2=24.55; P<0.001), respectively. The progression-free survival (PFS) of the second-line regimens (2ndPFS) was 17.7 vs. 9.0 vs. 9.2 vs. 4.6 months (χ2=22.74; P<0.001), respectively, among which patients in the PI/IMiD and PI+IMiD groups had comparable 2ndPFS (χ2=1.76; P=0.923). Patients with high-risk cytogenetic abnormalities (HRCAs) achieved the longest 2ndPFS of 22.0 months in the immunotherapy group (χ2=15.03; P=0.002). Multivariate analysis suggested that immunotherapy (HR=0.11, 95%CI 0.05-0.27), achievement of efficacy of partial response or better (HR=0.47, 95%CI 0.34-0.66), and non-aggressive relapse (HR=0.25, 95%CI 0.17-0.37) were independent prognostic factors of 2ndPFS. Conclusion: In this real-world study, immunotherapy was associated with a more favorable efficacy and PFS for first-relapsed MM patients after BCD treatment, with similar outcomes in patients with HRCAs.

[The 492nd case: recurrent thrombosis, thrombocytopenia].

A 43-year-old female patient was admitted with recurrent thrombosis for more than 2 years and thrombocytopenia for more than 1 year. Both arterial and venous thromboses developed especially at rare sites even during anticoagulation therapy such as cerebral venous sinus thrombosis. Antinuclear antibody, anti-ENA antibody and antiphospholipid antibody were all negative. Platelet count elevated to normal after high dose glucocorticoid and intravenous immunoglobulin (IVIG). Immune thrombocytopenia was suspected. When 4 grade thrombocytopenia recurred, intravenous heparin, rituximab 600 mg, IVIG and eltrombopag were administrated. After 3 weeks, thrombocytopenia did not improve, and new thrombosis developed instead. Screening of thrombophilia related genes revealed PROS1 gene heterozygous mutation and MTHFR TT genotype. Low amount of serum IgG κ monoclonal protein was detected. Heparin-induced thrombocytopenia was differentiated and excluded. Finally, serum negative antiphospholipid syndrome was considered the most likely diagnosis. Dexamethasone 20 mg/day × 4 days combined with sirolimus 2 mg/day was prescribed. The patient was discharged with low molecular weight heparin. At one month, her headache was greatly relieved. The platelet count raised to 20-30×109/L, and no new thrombosis or bleeding was reported.

[Clinicopathological features and prognosis of primary mediastinal large B-cell lymphoma: a series of sixty cases].

Objective: To investigate the clinicopathological features and prognostic factors of primary mediastinal large B-cell lymphoma (PMBL). Methods: The clinical data of 60 patients with PMBL including 44 biopsy cases and 16 consultation cases from September 2000 to November 2019 in the Department of Pathology, China-Japan Friendship Hospital (14 cases) and Peking Union Medical College Hospital (46 cases) were enrolled. Pathologic features, immunophenotype, immunoglobulin (Ig) gene rearrangement and microRNA expression profile were retrospectively studied. Results: Of the 60 patients, 23 were males and 37 were females, age ranged from 15 to 64 years (median 28 years). Immunohistochemical staining showed that the tumor cells were positive for pan-B cell antigens, CD30 (77.4%, 24/31), CD23 (73.1%, 19/26), MUM1 (45.8%, 11/24), Ki-67 index ≥70 % (90.6%, 29/32). EBER in situ hybridization was analyzed in 21 PMBL, only one case (4.8%) was positive. Ig gene rearrangement was performed in 20 cases, and seven were positive (35.0%). MicroRNA gene expression profiles were analyzed in seven cases of PMBL and nine cases of diffuse large B-cell lymphoma, and there were 33 microRNAs with significant difference (P<0.05). Univariate analysis indicated that the poor prognostic factors included serum lactate dehydrogenase (LDH) level,International Prognostic Index (IPI) score ≥3, stages Ⅲ-Ⅳ, chemotherapy not combined with rituximab and MUM1 positivity (P<0.05). Multivariate analysis showed that the treatment combined with rituximab was independently related to prognosis (P<0.05). Conclusions: PMBL is different from diffuse large B-cell lymphoma in clinicopathologic features, immunophenotypic presentation and molecular features. The prognostic factors, molecular genetics and immunological characteristics reveal that this study has enriched our understanding of the biology of PMBL, thus providing evidence and strategies for treatment.

[The clinical manifestations and risk factors in primary antiphospholipid syndrome with thrombocytopenia].

Objectives: To cross-sectionally analyze the clinical characteristics of primary antiphospholipid syndrome (PAPS) patients with thrombocytopenia, risk factors associated with thrombocytopenia, and risk of symptom recurrence in these patients. Methods: The inpatients with PAPS were retrospectively analyzed in Peking Union Medical College Hospital from 2009 to 2019. Using the collected clinical and laboratory data, the clinical characteristics and risk of symptom recurrence in the PAPS patients with thrombocytopenia were compared with those in the PAPS patients with normal platelet counts. Univariate and multivariate logistic regression analyses were performed to screen the risk factors for thrombocytopenia. Results: In this study, 127 patients with PAPS were enrolled, of which 36 (28.3% ) had thrombocytopenia, with a median age of 38 years, and 63.9% were female. In the thrombocytopenia group, the average platelet count was (58.9±27.0) ×10(9)/L, and the prevalence of thrombosis and morbid pregnancy was not significantly different from that in the normal platelet group. However, the thrombocytopenia group had higher incidence rate of autoimmune hemolytic anemia (19.4% vs 3.3% ) , livedo reticularis (16.7% vs 3.3% ) , chronic kidney disease (25% vs 8.8% ) and antiphospholipid antibodies triple positiveness (61.1% vs 37.4% ) , lower complement levels (C3 of 0.87 g/L vs 1.07 g/L, C4 of 0.12 g/L vs 0.18 g/L, P<0.05) , and higher adjusted Global APS Score (median score of 13 vs 9, P=0.037) than the normal platelet group. In multivariate logistic regression analysis, hypocomplementemia (OR value 5.032, 95% CI 3.118-22.095) is an independent risk factor for thrombocytopenia. Conclusions: In patients with PAPS, thrombocytopenia is mostly mild to moderate. Hypocomplementemia may be the independent risk factor for thrombocytopenia in PAPS patients. The PAPS patients with thrombocytopenia may have a higher risk of symptom recurrence.

[Orthopedic treatment of musculoskeletal disorders in hemophilic patients].

Objective: To study the orthopedic treatment strategy for hemophilia complicated with musculoskeletal disorders as well as the peri-operative consumption of clotting factor. Methods: Total 338 orthopedic surgeries were performed for 261 patients, average age of 30.6 y (6-65 y) , with hemophilia between January 1996 and December 2019 at our institute. Two hundred and twenty-six patients presented with bleeds within the joints. Sixty-one patients presented with intramuscular bleeds, 45 presented with hemophilic pseudotumors, and six presented with miscellaneous complaints. Strategy of clotting factor replacement therapy was designed as per differences in the level of the operation procedure. Information regarding clinical manifestation, operative strategy, clotting factor consumption, and re-operation for complications was retrospectively recorded. The costs for multiple joint procedure and single joint procedure were studied. Results: We found that 270 of the 338 surgical procedures were major surgical procedures (79.9%) . There were 203 procedures of joint arthroplasty (60%) . Fourteen patients underwent reoperations for local recurrence (4.2%) . The average factor Ⅷ consumption before the surgery was 44.4 ± 8.1 IU/kg. The average FⅧ consumption within postoperative 2 weeks was 40 962 IU (647±177 IU/kg) . Seven type A hemophilic patients developed F Ⅷ inhibitor following the surgical procedure, with an average level of 13.7±11.2 BU/mL. Sixty-eight patients underwent multiple joint procedures under one anesthesia session (26%) . There was no significant difference in the factor consumption between the multiple joint procedure and single joint procedure. Conclusions: Surgical treatment was found to be effective for hemophilic arthropathy and lesion of the musculoskeletal apparatus, with the clotting factor replacement therapy. Multiple joint procedures under one anesthesia were more cost effective for patients with hemophilia, with less factor consumption than staged single joint procedure.

[A pediatric-inspired regimen for adolescents and young adults with Philadelphia chromosome-negative acute lymphoblastic leukemia: results from a single center].

Objective: To investigate the efficacy of using a pediatric-inspired regimen for adolescents and young adults (AYA) with Philadelphia chromosome-negative (Ph(-)) acute lymphoblastic leukemia/lymphoblastic lymphoma (ALL/LBL) at a single center in China. Methods: Clinical data of 71 consecutive newly diagnosed AYA patients with Ph(-) ALL/LBL on a pediatric-inspired regimen in Peking Union Medical College Hospital from January 2012 to November 2018 were retrospectively analyzed. Results: Median age at diagnosis was 20 years (range: 15-38) , and 46 patients (64.8%) were male. Forty-nine (69.0%) had B-ALL/LBL. Among 62 ALL patients, 22 (35.5%) were high-risk. Complete remission rate was 93.0%. At follow-up with a median time of 44 months, the estimated 5-year disease-free survival (DFS) and overall survival (OS) was 56.3% and 64.3%, respectively. There was no significant difference in 5-year OS between allogeneic hematopoietic stem cell transplantation group and the continuous chemotherapy group after completion of 4 courses of chemotherapy. The 5-year DFS and OS for the non-high-risk group was 63.1% and 73.7%, respectively, which were significantly higher than 32.0% and 44.4% for the high-risk group, respectively (P<0.001) . Conclusions: The use of pediatric-inspired regimen for AYAs with Ph(-) ALL/LBL was feasible and effective.

[Purpura fulminans related to paroxysmal nocturnal haemoglobinuria: a case report and literatures review].

Objective: To improve the understanding of the rare clinical presentation and management of purpura fulminans (PF) in patients with paroxysmal nocturnal haemoglobinuria (PNH). Methods: A case of PF occurring in PNH is reported, while the related literature review is conducted. Results: A 49-year-old male patient suffered from one-week history of fever, greenish-brown colour urine, multiple well demarcated and painful purpura of the head and neck. He had been reported to have two thromboembolic events during the 22-year course of PNH. Skin biopsy displayed classic PF features. Laboratory testing showed a high PNH clone, intravascular hemolysis and coagulation system changes. After sufficient anticoagulation and short course of glucocorticoid therapy, the clinical conditions were improved correspondingly. During a follow-up period of 6 month, there was no recurrence of thrombosis. Conclusion: PF should be considered in PNH patients with unexplained, quickly developed painful purpura. Extensive work-up should be performed to find out other potential thrombophilic risk factors after diagnosis of PF. Early diagnosis, adequate anticoagulation therapy and control hemolysis were essential to PF treatment occurring in PNH. The survival of patients and the qualities of life can be improved. The PNH clone detection is needed to evaluate the status of procoagulation and predict the risk of recurrent thrombosis.

[Lymphomatoid gastropathy: one case report and literatures review].

Objective: To report the first case of lymphomatoid gastropathy in China, and to demonstrate the clinical characteristics, diagnostic approach, treatment and prognosis in this kind of patients. Methods: One patient was diagnosed as lymphomatoid gastropathy at Peking Union Medical College Hospital, and her clinical characteristics, lab data, treatment and follow-up outcomes were reviewed. Results: A case of a 51-year-old female was presented, who underwent esophagogastroduodenoscopy (EGD) due to slight epigastric discomfort. EGD revealed multiple ulcers and erosions. Biopsies showed atypical lymphocytes infiltration with CD3(+), CD56(+), CD20(-), CD8(-), TIA(+), Granzyme B(-) and Ki-67 (75%). Epstein-Barr virus-encoded RNA in situ hybridization was negative. Four months later, repeated EGD examination showed regression of the lesions without specific treatment. Conclusion: Lymphomatoid gastropathy was a unique disease entity mimicking NK/T-cell lymphomas in pathology, with the quite different profile of treatment and prognosis. It's important to consider this issue during the differential diagnosis to avoid any excessive treatment.

[Posaconazole as primary prevention of fungal infection in intensive immunosuppressive therapy for severe aplastic anemia].

Objective: To Evaluate the efficacy and safety of posaconazole as primary prevention of invasive fungal disease (IFD) in patients with severe aplastic anemia (SAA) treated with anti-thymus/lymphocyte immunoglobulin (ATG/ALG) combined with cyclosporine intensive immunosuppressive therapy (IST). Methods: A retrospective analysis of clinical data of 58 SAA patients who received IST of anti-thymocyte immunoglobulin combining cyclosporine and antifungal prophylaxis during April 2013 to May 2017 in Peking Union Medical College Hospital was performed. The patients were divided into posaconazole prophylaxis group and the control group (itraconazole or fluconazole). The disease characteristics, IFD prevention effect and adverse drug reaction, curative effect and prognosis of the two groups were compared. Results: Posaconazole was used to prevent fungal infection in 20 patients. The other 38 patients were used as the control group. Retrospective analysis showed comparable characteristics (gender, age, disease severity, etiology, interval between the onset of disease to treatment, ATG/ALG type) of both groups. The incidence of IFD were 0 and 15.8% in posaconazole prophylaxis group and the control group, respectively (P=0.084). In the control group, there were 6 cases diagnosed as IFD. Of them, 2 were confirmed, 2 suspected and 2 not identified. Five of the 6 cases were pulmonary infection, 1 bloodstream infections. Of the 6 IFD cases, 5 were very severe aplastic anemia (VSAA). There was no obvious adverse reaction in posaconazole prophylaxis group. Conclusion: Posaconazole is safe and effective for primary prevention of fungal infection of SAA patients receiving IST, especially for the VSAA.

[Clinical characteristics of intravascular large B cell lymphoma: a single-center retrospective study].

Objective: To investigate clinic-pathological characteristics, diagnosis, treatment and prognosis of intravascular large B cell lymphoma (IVLBCL) in China. Methods: Clinical and pathological records were analyzed from 12 IVLBCL patients diagnosed between Jan 2010 to Jun 2016. Kaplan-Meier method was used to estimate overall survival (OS), and univariate analysis was performed to identify prognostic factors. Results: A series of 12 patients with IVLBCL (median age, 53.8 years; range, 32-76 years; 6 males and 6 females) was reviewed. Fever was the most common symptom (10/12), respiratory symptoms (cough, pleural effusion, dyspnea, 50%) and hemophagocytic lymphohistiocytosis (50%) were frequently observed, and only 12 patients had neurological symptom. All patients had elevated lactic dehydrogenase and serum ferritin. International Prognostic Index score was high in 75% of total patients. All patients had extra-nodal involved, pulmonary (6/12) and bone marrow (4/12) were frequently involved. Large lymphoid cells within vessel lumina or sinuses were observed in all patients. These cells were large, with scant cytoplasm, vesicular nuclei, and one or more nucleoli, and the structures of vessels and sinus were reserved. CD20 and CD79a were positive in all cases. 11patients received rituximab combined CHOP regimen chemotherapies, overall response rate (ORR) was 90.1%, and complete response rate was 66.7%. Median survival time and median progression time were not reached after a median follow-up of 20 months. Univariate analysis revealed that no clinical characters were associated with OS. Conclusion: As a rare variant of DLBCL, IVLBCL presented with pulmonary involved frequently, and trans-bronchial lung biopsy had good positive rates. Rituximab contained chemotherapy was the backbone for IVLBCL.

[Evaluation of clinical characteristics, MYD88(L265P) mutation, CXCR4(WHIM) mutation and prognosis in Waldenström macroglobulinemia: A single center retrospective study of 93 patients].

Objective: To evaluate the clinical characteristics, MYD88(L265P) mutation, CXCR4(W)HIM mutation and prognosis in patients with Waldenström macroglobulinemia (WM). Methods: The clinical characteristics, International Prognostic Scoring System for symptomatic WM (WPSS) , and overall survival (OS) were retrospectively assayed in 93 patients with newly diagnosed WM at Peking Union Medical College Hospital during January 2000 to August 2016. The MYD88(L265P) mutation and CXCR4(W)HIM mutation were tested among 34 patients. Results: The median age of the 93 patients was 64 years (range, 33-85 years) with a male-to-female ratio of 2.44. According to WPSS, we included 16 (17.2%) low-risk, 44 (47.3%) intermediate-risk and 33 (35.5%) high-risk patients. Eight patients had secondary amyloidosis. With a median follow-up of 44 (1-201) months, the median OS was 84 months. Cox regression multifactor analysis showed WPSS risk group (HR=2.342, 95% CI 1.111-4.950, P=0.025) , whether patients had secondary amyloidosis (HR=5.538, 95% CI 1.958-15.662, P=0.001) and whether patients received new drugs (HR=3.392, 95% CI 1.531-7.513, P=0.003) were independent factors associated with OS. We have investigated the presence of the MYD88(L265P) and CXCR4(WHIM) mutation in 34 patients and found that MYD88(L265P) mutation was occurred in 32 patients (94.1%) and CXCR4(WHIM) mutation was occurred in 8 patients (23.5%). Seven of 8 patients who harbored CXCR4(WHIM)-mutated also exhibited the MYD88(L)265P mutation. Patients with MYD88(L265P)CXCR4(WHIM) vs MYD88(L265P)CXCR4(WT) presented with more severe anemia, lower platelet level, higher M protein level and more hyper-viscosity syndrome. Conclusion: WPSS risk group, whether patients had secondary amyloidosis or received new drugs are independent factors for OS in WM. MYD88(L265P) and CXCR4(WHIM) mutation, the most common somatic variants in WM, often occur together and impact the clinical presentation.

[The 455th case: swollen leg, jaundice and mental disturbance].

A 17-year-old young man with a history of swollen leg and intermittent jaundice was presented to Peking Union Medical College Hospital with acute fever and mental disturbance. He developed deep venous thrombosis, acute myocardial infarction and plantar skin necrosis during the past four years, and was presented with an acute episode of fever, thrombocytopenia, acute kidney injury, acute myocardial infarction, mental disturbance, and obstructive jaundice. Laboratory tests showed schistocytes on peripheral blood smear.High titer of antiphospholipid antibodies was detected.Strikingly, the activity of a disintegrin and metalloprotease with a thrombospondin type 1 motif, member 13 (ADAMTS13)was significantly decreased without the production of inhibitors. Images indicated stenosis of the common bile duct, common hepatic duct, and cystic duct, which caused dilation of bile ducts and the gall bladder. Corticosteroids and anticoagulation therapy were effective at first, but the disease relapsedonce the corticosteroids tapered down. Plasma exchange was administrated for 17 times, which was effective temporarily during this episode. Methylprednisolone pulse therapy, intravenous immunoglobulin, rituximab, anticoagulation therapy, and bile drainage, were all tried but still could not control the disease. The patient's family agreed to withdraw treatment after he developed septic shock.

Impact of pretransplant induction therapy on autologous stem cell transplantation for patients with newly diagnosed POEMS syndrome.

POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy and skin changes) is a rare plasma cell dyscrasia sometimes treated with a haematopoietic cell autotransplant. We analyzed data from 138 subjects with newly diagnosed POEMS syndrome receiving a autotransplant at our center. Thirty-two subjects with severe end-organ dysfunction ineligible for immediate autotransplant received pretransplant therapy, which made a subsequent autotransplant feasible. Pretransplant therapy resulted in vascular endothelial growth factor (VEGF) remissions in 15 (47%). Thirty-three transplant recipients (24%) had early posttransplant complications. Risk factors for these complications identified through multivariate analysis included age >50 years (odds ratio (OR) 2.79, 95% confidence interval (CI) 1.09-7.14; P=0.033), time from symptom onset to transplant >5 years (OR 4.71, 95% CI 1.10-20.18; P=0.037) and pleural effusion (OR 3.39, 95% CI 1.26-9.12; P=0.016). Subjects receiving pretransplant therapy had fewer early complications than those who did not (OR 0.17, 95% CI 0.04-0.71; P=0.015), especially in subjects with a VEGF remission (OR 0.05, 95% CI 0.01-0.49; P=0.010). Autotransplants resulted in hematological remission in 60 (50%), VEGF remissions in 76 (72%) and improvements in other organ functions (65-90%). The 5-year progression-free survival (PFS) and overall survival were 76% (95% CI 64-84%) and 94% (95% CI 87-97%), respectively. Hematological (5-year PFS 83 vs 66%, P=0.008), VEGF (5-year PFS 79 vs 57%, P=0.021) remissions and especially both (5-year PFS 95 vs 61%, P=0.004) were associated with better PFS.

[Analysis of the safety and efficacy of 60 mg·m-2·d-1daunorubicin combined with standard dose of cytarabine as induction therapy in acute myeloid leukemia patients under 65 years old].

Objective: To evaluate the long- term safety and efficacy of high- dose daunorubicin(DNR)(60 mg·m-2·d-1)combined with standard dose of cytarabine(DA)as induction therapy in patients under 65 years old with newly diagnosed acute myeloid leukemia(AML). Methods: The complete remission(CR)rate, disease free survival(DFS), overall survival(OS)and side effects of therapy were retrospectively assayed in 116 patients with newly diagnosed AML who were younger than 65 years old and received daunorubicin(60 mg · m-2·d-1)combined with cytarabine(Ara- C 200 mg ·m-2·d-1)as induction therapy at Peking Union Medical College Hospital during July 2012 to February 2016. Results: Of 116 patients, 78 cases(67.2%)achieved CR after first course of induction treatment, 94(81.0%)achieved CR after two courses of induction, and early death occurred in only 3 patients(2.6%)during the first course of induction treatment. Only 1 patient had asymptomatic decreased ejection fraction after 6 months of induction treatment. Eighty nine patients received 1 to 4 courses of consolidation. With a median follow-up of 24(1-46)months, the median DFS was 25 months and median OS was not achieved yet. Cox regression multifactor analysis showed genetics risk groups was the only risk factor for DFS(HR=0.258, 95% CI 0.100- 0.664, P=0.005), while genetics risk groups(HR=0.309, 95% CI 0.126- 0.756, P=0.010)and whether patients received more than one cycle of high dose of Ara-C as consolidation therapy(HR= 0.370, 95% CI 0.179- 0.765, P=0.007)were independent factors associated with OS. Conclusions: In young adults with AML, intensifying induction therapy with a high daily dose of daunorubicin(60 mg/m2)could improve the rate of complete remission without obvious side effects.

[Autologous peripheral blood hematopoietic stem cell transplantation for patients with primary light chain amyloidosis: experience of 31 cases in a single center].

OBJECTIVE: To evaluate the efficacy and safety of autologous peripheral blood hematopoietic stem cell transplantation (ASCT) for patients with primary light chain (AL) amyloidosis. METHODS: Clinical data, hematological and organ response, safety and survival status of 31 patients with AL amyloidosis who had received ASCT from January 2009 to June 2015 were retrospectively analyzed. RESULTS: Among 31 patients, there were 18 males and 13 females with the median age of 55 (range, 43-66) years old. Involvement of 1 organ was presented in 20 patients. 80.6% patients were defined as Mayo stage 1. The median time from diagnosis to ASCT was 3 (range, 0.5-26) months. The median time to neutrophil and platelet engraftment was 11 (range, 9-12) days and 11 (range, 8-14) days, respectively. No one patient had transplantation related death. Among 27 evaluable patients, overall best hematological response was 85.2% with complete response of 63.0% and very good partial response of 7.4%. The median time to the best hematological response was 4 (range, 1-21) months. 59.2% patients archived organ response and the median time to organ response was 8 (range, 3-18) months. After the median follow up time of 21 months, one patient had died and three patients had progressed. Therefore, the estimated 3 years progress free survival and overall survival was 92.8% and 96.4%, respectively. CONCLUSIONS: ASCT was an effective and safe treatment for patients with primary AL amyloidosis in early stage.

Comparison of vindesine and prednisone and cyclophosphamide, etoposide, vindesine, and prednisone as first-line treatment for adult Langerhans cell histiocytosis: A single-center retrospective study.

OBJECTIVE: We compared the efficacy and clinical outcomes of vindesine and prednisone (VP) and cyclophosphamide, etoposide, vindesine, and prednisone (CEVP) regimens as first-line treatment for multisystem (MS) or multifocal single system (SS-m) adult Langerhans cell histiocytosis (LCH). METHOD: Clinical features, treatment response, and survival of adults with Langerhans cell histiocytosis treated at our center from January 2001 to January 2015 were reviewed retrospectively. RESULTS: Forty-five adult MS or SS-m LCH patients were treated (N=31, CEVP group; N=14, VP group). Both treatment groups had similar gender distributions, patient ages, and extent of disease. The non-active disease rate for both groups was 70.0% and 64.3% (P=0.775), respectively. Median follow-up was 74.9 (range: 2.8-183.6) months and recurrence rates were 71.0% and 78.6% (P=0.593), respectively. The need for second-line therapy was 64.5% and 71.4% (P=0.649), respectively, and mortality rates were 9.7% and 15.4% (P=0.586), respectively. Neutropenia occurred in 48.4% of CEVP-treated patients and 7.1% of VP-treated patients (P=0.008). CONCLUSIONS: CEVP or VP regimens for the treatment of adult SS-m or MS LCH showed similar efficacies, and both regimens were associated with high disease recurrence and the need for second-line therapy.