scholarly journals A Case of Acquired von Willebrand Syndrome Complicated by Acute Myelomonocytic Leukemia

2021 ◽  
pp. 1152-1158
Author(s):  
Masahiko Fukatsu ◽  
Hiroshi Ohkawara ◽  
Hiroshi Takahashi ◽  
Hirotaka Mori ◽  
Mai Yaginuma ◽  
...  
Keyword(s):  
Stem Cell ◽  
Complete Remission ◽  
Stem Cell Transplantation ◽  
Remission Induction ◽  
Acquired Von Willebrand Syndrome ◽  
Remarkable Improvement ◽  
First Case ◽  
Acute Myelomonocytic Leukemia ◽  
Von Willebrand ◽  
Myelomonocytic Leukemia

We here report a 21-year-old male who presented with acute myelomonocytic leukemia (AMML) associated with acquired von Willebrand syndrome (AVWS). To our knowledge, this is the first case of AVWS caused by AMML. In our case, following remission-induction chemotherapy combined with idarubicin and cytarabine, the patient showed remarkable improvement of bleeding symptoms due to AVWS. Moreover, after an allogeneic stem cell transplantation from a sibling donor, both AMML and AVWS maintain complete remission.

scholarly journals Acquired von Willebrand Syndrome associated to secondary IgM MGUS emerging after Autologous Stem Cell Transplantation for AL Amyloidosis

2017 ◽  
Vol 9 (1) ◽  
pp. e2017034 ◽  
Author(s):  
Victor H Jimenez-Zepeda ◽  
Hina Qamar ◽  
Adrienne Lee ◽  
Karen Valentine ◽  
Leslie Skeith
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Autologous Stem Cell Transplantation ◽  
Conditioning Regimen ◽  
Al Amyloidosis ◽  
Acquired Von Willebrand Syndrome ◽  
First Case ◽  
History Of ◽  
Von Willebrand

Acquired von Willebrand syndrome (AVWS) is a rare hemorrhagic disorder that occurs in patients with no prior personal or family history of bleeding. Here, we describe a case of AVWS occurring after autologous stem cell transplantation (ASCT). Interestingly, AVWS developed after bortezomib-based induction and conditioning regimens. Recent evidence suggests that the proximity of the bortezomib therapy to the collection of stem cells with consequent depletion of regulatory T cells after the conditioning regimen could explain some of the unusual autoimmune complications reported in patients receiving bortezomib prior to ASCT. In addition, this patient developed a secondary MGUS post-ASCT, which may have also contributed to the AVWS. To the best of our knowledge, this is the first case of post-ASCT AVWS reported. Prospective data is needed to better elucidate the mechanisms by which these unusual complications occur in patients receiving bortezomib prior to ASCT.

Rapid Cyclosporine Tapering and Isotretinoin Usage after Unrelated Donor Hematopoietic Stem Cell Transplantation in Juvenile Myelomonocytic Leukemia.

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 5188-5188
Author(s):  
Keon Hee Yoo ◽  
Soo Hyun Lee ◽  
Ki Woong Sung ◽  
Hong Hoe Koo ◽  
Hye Lim Jung
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Complete Remission ◽  
Stem Cell Transplantation ◽  
Acute Gvhd ◽  
Juvenile Myelomonocytic Leukemia ◽  
Hematopoietic Stem ◽  
Differentiating Agent ◽  
Myelomonocytic Leukemia ◽  
Disease Free

Abstract Juvenile myelomonocytic leukemia (JMML) is a rare type of childhood leukemias, and allogeneic hematopoietic stem cell transplantation (HSCT) is known to be the only way to cure the disease. Unfortunately, relapse is still the most frequent cause of treatment failure after transplant in JMML. We investigated the feasibility of inducing graft versus leukemia (GVL) effect and the use of a differentiating agent even after unrelated HSCT in children with JMML. Seven consecutive patients with JMML underwent unrelated HSCT at a median age of 17 months. The sources of grafts were bone marrows (n=3) or HLA 1- or 2-antigen mismatched cord bloods (n=4). Only 3 of the 7 patients were in complete remission before transplantation. Intravenous busulfan, cyclophosphamide, and etoposide were used as preparative agents except in one who was conditioned with TBI-based regimen. Cyclosporine was used universally for GVHD prophylaxis with additional use of short-term methotrexate in bone marrow transplants and of methyl-prednisolone in cord blood transplants. Cyclosporine was tapered rapidly from around 1 month post-HSCT and isotretinoin (75–100 mg/m2/day) was used in selected patients who have any risk factors of relapse. Cyclosporine blood levels were 247.8±91.1, 146.6±104.2, and 88.8±52.6 ng/mL at 1, 2, and 3 months post-transplant, respectively. There was no grade 3 or 4 acute GVHD and only 2 patients developed grade 2 acute GVHD which was improved without additional treatment. Chronic GVHD was developed in 3 (1 limited, 2 extensive) of the evaluable 5 patents, which was all resolved after combined use of immune suppressive agents. Initial chimeric status analysis at 1 month revealed complete donor chimerism (CC) in 4 patients, mixed chimerism (MC) in 2 and autologous recovery (AR) in one. One of the patients with MC and the one with AR were in disease-free status. One patient whose chimeric status changed from CC to MC eventually relapsed. One patient with initial MC with residual disease turned to CC with complete remission. Another patient with initial MC but with no evidence of disease is on treatment with isotretinoin without relapse for 3 months even with persistent MC. The patient with AR relapsed early after transplant. Five patients are alive relapse-free and disease-free with a median follow-up of 16 months after transplant. The Kaplan-Meier probability of event-free survival was 66.7%. We suggest that GVL induction strategy with concomittant use of a differentiating agent might have a role to suppress leukemic relapse in JMML.

scholarly journals Feasibility and Outcome of Hematopoietic Stem Cell Transplantation Combined with Decitabine for Children with Juvenile Myelomonocytic Leukemia

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 5869-5869
Author(s):  
Huaying Liu ◽  
Chunfu Li ◽  
Yuelin He
Keyword(s):  
Bone Marrow ◽  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Complete Remission ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Juvenile Myelomonocytic Leukemia ◽  
Unrelated Donor ◽  
Hematopoietic Stem ◽  
Myelomonocytic Leukemia

Abstract Background Juvenile myelomonocytic leukemia(JMML) is a rare clonalmyelodysplastic/myeloproliferative disorder that occurs during infancy and early childhood with poor prognosis. Chemotherapy has not been found to be dffective, and Hematopoietic stem cell transplantation(HSCT) is currently the only curative treatment for JMML. Relapse and engraftment failure are the major causes of HSCT failure in JMML. Patients and method We report the outcomes of 4 patients with JMML who received HSCT combined with Decitabine between 2014-2015. Patient median age was 2 years(range,1-3years), and 3 were boys and 1 girl. Decitabine was given before and after the HSCT for one time(20mg/m2.d X 5d、10mg/m2 .d X 5d). Before HSCT, all the patients received mild chemotherapy(three or four course). The bone marrow evaluations of all the patients before HSCT were complete remission(CR). Two patients received human leukocyte antigen(HLA)-matched HSCT from unrelated donors, and two patients received haploidentical HSCT from parents followed by unrelated cord blood transplantation(UCB). Conditioning regimen of Unrelated donor-PBSCT was Busulfan+fludarabine+Thiotepa+Thymoglobuline, and the conditioning of haplo-HSCT was Busulfan+fludarabine+Cytarabine+Thymoglobuline. The number of nucleated cells of HLA-matched HSCT was 8×108/kg. The number of nucleated cells of Haplo-HSCT was 47.2×108/kg、61.26×108/kg , respectively, and the number of nucleated cells of UCB was 7.23×107/kg、9.4×107/kg, respectively. GVHD prophylaxis was based on post-transplant high-dose cyclophosphamide(PTCy, 50mg/kg on days +3 and +4) combined with mycophenolate plus cyclosporine A or tacrlimus. Results: The median follow-up was 21 months(range,11-27 months). The overall survival(OS) and the Disease free survival(DFS) both were 100%, All the patients got 100% engraftment(Unrelated-donor stem cell engrafted and Haploidentical-donor stem cell engrafted in 2 and 2 patients , respectively). None of the patients developed relaps, the bone marrow evaluations were complete remission(CR) after HSCT. The most common toxicities were infection with neutropenia(100%, n=4), The cumulative incidences of acute GVHD gradesII-III and CMV infection were 50% and 75% respectively. Conclusion: The combination of decitabine and HSCT shows encouraging results with highly effective and less toxicity for JMML. The futhuer study should be developed in the future. Disclosures No relevant conflicts of interest to declare.

Association of Acquired Von Willebrand Syndrome with Primary (AL) Amyloidosis.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 4078-4078
Author(s):  
Cynthia A. Kos ◽  
Jennifer E. Ward ◽  
Karim Malek ◽  
Vaishali Sanchorawala ◽  
Daniel G. Wright ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Autologous Stem Cell Transplantation ◽  
Amyloid Fibrils ◽  
High Dose ◽  
Al Amyloidosis ◽  
Factor X ◽  
Acquired Von Willebrand Syndrome ◽  
Von Willebrand

Abstract Easy bruising is a common clinical symptom in primary (AL) amyloidosis, and can occur through multiple mechanisms. Infiltration of amyloid fibrils into thin-walled capillaries leads to mechanical fragility predisposing to petechiae and purpura, with periorbital “raccoon-eye” purpura being pathognomonic of AL amyloidosis. Another mechanism predisposing to bleeding in AL amyloidosis is adsorption of coagulation factors to amyloid fibrils. In 1977, Furie et al. reported (New England Journal of Medicine) that elevation of the clotting times is most commonly due to deficiency of factor X, and other investigators have since reported deficiencies of factors II, V, IX, or XIII. We have demonstrated that remission of the underlying plasma cell dyscrasia after high dose melphalan chemotherapy and autologous stem cell transplantation can lead to remission of the acquired factor X deficiency (Choufani et al., Blood 2001). From 2000–2004, four amyloidosis patients presented to Boston University Medical Center with bleeding and a prolonged activated partial thromboplastin time (aPTT), but with no such factor deficiency. Instead, they were found to have abnormal von Willebrand ristocetin cofactor (vWF:RCo) and/or factor VIII (FVIII:C) activities, with normal vWF antigen (vWF:Ag), consistent with a functional defect in von Willebrand factor (vWF). None of the patients had a prior history or family history consistent with congenital von Willebrand’s disease, thus they were diagnosed with acquired von Willebrand syndrome (AvWS). AvWS has most often been reported in association with other lymphoproliferative or myeloproliferative disorders. The aPTT was prolonged in three out of the four cases. Loss of high molecular weight multimers (HMWM) was observed in two of the four cases. Two of the patients were treated with high-dose intravenous melphalan followed by autologous stem cell transplantation and achieved remission of their underlying plasma cell disease; in addition, the bleeding diathesis ceased and the coagulation parameters normalized, indicating reversal of the AvWS with effective treatment of AL amyloidosis.

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