Audit of clinical management of von Willebrand disease during 1997 at a single institution and review of treatment patterns between 1980 and 1997

Abstract

In 1997 the UK Haemophilia Centre Directors Organization published the guidelines for diagnosis and management of von Willebrand disease (vWD). The guidelines stated that desmopressin (DDAVP) should be used in type 1 and type 2N vWD, that it might be useful in other types 2 vWD and that it is ineffective in type 3 vWD. If patients are unresponsive to DDAVP or if it is contraindicated, the treatment of choice is clotting factor concentrates (CFC). In the light of these guidelines, we audited our practice for 1997. Furthermore, we undertook a retrospective review of the changing patterns of treatment of vWD between 1980 and 1997. During 1997, 10 patients with vWD received DDAVP and another 30 patients were treated with CFC (a total of 1.2 million IU): Haemate P (Centeon, Germany) and/or 8Y (BPL, Elstree, UK). Few patients had clear contraindications to DDAVP, but several patients with type 1 and 2 vWD received CFC on the basis of age or reduced levels of von Willebrand factor - where DDAVP was considered suboptimal for adequate haemostasis. However, this assumption was made without a preliminary test dose to assess the response to DDAVP. The analysis of treatment of vWD for the past 17 years showed that cryoprecipitate was discontinued from use in the early 1990s and that both DDAVP and CFC usage has been on the increase. In conclusion, the audit illustrated a general good adherence to guidelines but it highlighted the need for a DDAVP test before using CFC.

Management of von Willebrand disease: a survey on current clinical practice from the haemophilia centres of North America

“…Guidelines were recently developed for both the diagnosis and treatment of patients with vWD disease by the United Kingdom Haemophilia Centre Directors Organization [6, 7]. A retrospective review of adherence to these guidelines in which an audit of 10 patients treated with DDAVP and 30 patients treated with clotting factor concentrates was recently reported [8]. Both the use of DDAVP and of vWF‐containing FVIII concentrates were found to be steadily rising and in general, patients were treated according to the UK guidelines.…”

Acquired von Willebrand syndrome and lymphoid neoplasms: A review of malignancy management, and propositions of practical recommendations

“…For all AWS‐PRD, the use of antimyeloma drugs produced to an AWS‐ORR of 71.4% (20/28 patients) 13,19,22,23,25–37 (Table 3), including three ASCT used as the sole treatment. Notably, bortezomib steroid‐based regimens were used nine times with an AWS‐ORR of 66.7% (6/9), 19,22,23,25–27,29,30 including associations with thalidomide, 27 melphalan, 25 cyclophosphamide 26,27 and lenalidomide 30 .…”

“…30 For ASCT, nine lines of treatment included autologous haematopoietic stem cell transplantation (HSCT) with an AWS-ORR of 88.9%. [25][26][27]30,31,33,34 Autologous HSCT was performed immediately 33,34 or was preceded by induction therapy. Among the recently available antimyeloma drugs, daratumumab was successfully used twice, 23,25 and carfilzomib was described once with an unclear overall response.…”

Haemate®P von Willebrand factor/factor VIII concentrate: 25 years of clinical experience

“…Because of its VWF content and safety and tolerability profile, Haemate P soon became established as an effective VWD treatment for those patients who could not be adequately treated with desmopressin [45]. Further evidence of the clinical efficacy of Haemate P was reported in the 1990s in VWD [52][53][54][55][56][57][58][59][60][61][62][63][64][65]. In a 1999 review of VWF-containing factor VIII concentrates, Berntorp concluded that Ôclinical studies and experience with Haemate P have been so successful that this concentrate has, for many years now, been considered the golden standard in replacement therapy for VWDÕ [43].…”